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The Future Looks Bright: Predictions for 2024



2023 drug approvals: After a down year, FDA signs off on a bounty of new meds, including 7 from Pfizer


In 2022, fueled by its powerhouse COVID products, Pfizer became the first company in the history of the biopharma industry to top $100 billion in annual revenue. What would the New York drugmaker do for an encore?


In 2023, Pfizer went from generating the most sales in the industry to gaining the most approvals.


With seven FDA nods in 2023, Pfizer had more than double that of any other company. It’s also more than twice as many as every drugmaker over each of the last three years. You need to go back to 2019, when Novartis scored six approvals, to find a company that approached what Pfizer accomplished in 2023.


Pfizer’s splurge included four approvals in a dizzying five weeks in May and June. And all but one of the seven products has been pegged by analysts as a potential blockbuster.

Two of the newly approved Pfizer treatments were acquired with the help of the firepower the company gained with its massive sales of COVID vaccine Comirnaty and antiviral Paxlovid. Pfizer’s new ulcerative colitis drug Velsipity came by way of the company’s 2022 acquisition of Arena Pharmaceuticals for $6.7 billion. New nasal spray migraine therapy Zavzpret arrived by way of Pfizer’s $11.6 billion buyout of Biohaven, which also brought soon-to-be blockbuster migraine drug Nurtec.


The new Pfizer products cover a wide swath of indications between multiple myeloma drug Elrexfio, OPKO-partnered growth failure treatment Ngenla, first-in-class alopecia therapy Litfulo, RSV vaccine Abryvso and COVID-19 antiviral Paxlovid, which gained a full approval.

Pfizer has never scored in one year with so many diverse products. In 2018, for example, when the company achieved four approvals, all of the blessings were for oncology drugs.

Martin Mackay, Ph.D., now the co-founder of Connecticut biotech RallyBio who formerly guided the R&D efforts at Pfizer, remembers being ridiculed in 2010 when he told Bloomberg Television that the company—which was in a minor lull at the time—and the industry in general were in a “golden age of drug discovery.”


These days, Mackay is getting the last laugh.


“I was watching what was coming through the pipeline,” Mackay said in a recent interview with Fierce Pharma. “I was watching all the new modalities that were at our fingertips—cell therapy, gene therapy, RNA, interference RNA, messenger RNA, neucleotides, gene editing—my thought then was that 10 years from now it’s gonna be terrific in terms of approvals.”   


Bounty of approvals in 2023

After 2022 produced just 37 new drug approvals—the fewest by the FDA since 2016—the United States regulator signed off on a bumper crop of 55 new drugs in 2023. The list was the second longest in FDA history, coming up just shy of the single-year record of 59 approvals, which was set in 2018. Since this annual report includes vaccine and biologics approvals, the total haul came to 66 new drugs and biologics.

Mackay expects the annual numbers to continue to grow over the next decade. Looking at a list of approvals from a decade or two ago, Mackay said he was struck by the wide variety of modalities on this year's list. 


"And interestingly, small molecules are still the most," Mackay said. "That doesn't surprise me either because they're proven agents. They haven't stayed the same. The technology to work on small molecules is just so different from when I started in the industry."   

One potential explanation for the difference between the amount of approvals between 2022 and 2023 were the COVID-related inspection delays which held up some approvals. Another potential reason for the dearth of FDA approvals in 2022 could have been the heat the agency took for its much-criticized 2021 nod for Biogen and Eisai’s Alzheimer’s disease drug Aduhelm.


Cancer, as usual, saw the most approvals in 2023 with 16, including four bispecifics in a span of as many months. Two bispecifics for multiple myeloma—Talvey from Johnson & Johnson and Elrexfio from Pfizer—made the grade. There also were two bispecifics approved for B-cell lymphomas—Columvi from Roche and Epkinly from AbbVie and Genmab. Talvey and Epkinly have been tabbed with blockbuster potential.


Gene Therapies

Two of the most significant green lights came on the same day, Dec. 8, and for the same indication, sickle cell disease. Vertex and CRISPR landed a historic thumbs up for Casgevy, which became the first drug to reach the market developed with the revolutionary CRISPR gene editing technology. Simultaneously, bluebird bio earned its nod for another gene therapy, Lyfgenia.


Bluebird priced its treatment at $3.1 million, compared to $2.2 million for Casgevy, raising eyebrows at the time. But good news came days after the approval from bluebird as it signed a reimbursement deal with a major insurer.


Two other pricey gene therapies made the grade, as well. The single-dose tab for BioMarin’s Roctavian, which treats hemophilia A, clocks in at $2.9 million. Sarepta broke through with a ballyhooed approval to treat Duchenne muscular dystrophy with Elevidys and priced it to match the hype at $3.2 million.


RSV Vaccines Make the Grade

Also making major news in 2023 was the long-awaited emergence of RSV vaccines. GSK scored first in May with Arexvy for people 60 and older, followed by Pfizer which gained two nods for Abrysvo. In July, Sanofi and AstraZeneca gained a blessing for their antibody, Beyfortus, to protect infants. By 2028, each of the products is expected to generate sales of between $1.3 and $1.7 billion.

The first year of rolling out the RSV antibody didn’t go smoothly, however. In late 2023, amid a shortage of Beyfortus, Sanofi and AZ were scrambling to ramp up manufacturing. The companies were taken to task by U.S. lawmakers, demanding answers for their inadequate preparations.


Other Key Approvals

In the closely watched Alzheimer’s disease space, Biogen and Eisai won a nod for their treatment Leqembi. The blessing came 19 months after the FDA rejected an advisory committee recommendation and signed off on Biogen’s ill-fated Aduhelm.

The FDA blessing of Leqembi even came by way of the same accelerated pathway that facilitated Aduhelm’s highly criticized green light. But there are several reasons to be optimistic about the launch of Leqembi as it posted superior trial results and—in contrast to Aduhelm—its nod was recommended unanimously by a panel of independent advisors, paving the way for a full approval.


On the flip side, the FDA rejected Eli Lilly’s touted Alzheimer’s treatment donanemab. But the company appears to be on track for approval in 2024.


Other big winners in ’23

Four companies—GSK, Biogen, Chiesi and UCB—secured three approvals each in 2023. The trio for UCB included two nods which came on the same day in October, for plaque psoriasis medicine Bimzelx and myasthenia gravis treatment Zilbrysq. The Belgium-based company also earned a green light for another myasthenia gravis drug with a different mechanism of action, Rystiggo.

Biogen’s winners were Qalsody in ALS and two partnered drugs—Alzheimer’s med Leqembi, which it shares with Eisai, and postpartum depression drug Zurzuvae, which it splits with Sage.

GSK scored for myelofibrosis drug Ojjaara, kidney disease treatment Jesduvroq and RSV vaccine Arexvy. Chiesi of Italy gained approval for Fulsuvez for a rare skin condition and two enzyme replacement therapies—Lamzede and Elfabrio.


Another company that excelled with two major approvals was Astellas. The Japanese company scored with Veozah, a breakthrough treatment for hot flashes and night sweats. It also won approval for Izervay, a treatment for geographic atrophy that it acquired in its $5.9 billion buyout of Iveric Bio in early 2023. Both Veozah and Izervay have been pegged to exceed peak sales of more than $2 billion as the company attempts to compensate for the looming patent cliff for prostate cancer drug Xtandi.


Editor's note: The numbers for our annual roundup typically vary slightly from the FDA's official list, because our report includes approvals for biologic therapeutics and vaccines but excludes diagnostic imaging agents, which are represented on the FDA's list.


Leqembi

Active ingredient: LecanemabDisease: Alzheimer’s disease

Sales estimate: $4.7 billion (2028)

Approved: Jan. 6

Companies: Eisai and Biogen  

The scoop: Eisai and Biogen's Leqembi nabbed a speedy approval in Alzheimer’s disease (AD) at the very start of 2023, following this up with a full approval in July while also being named as one of Time’s best inventions of the year. Eisai hopes the drug can reach megablockbuster territory and go some way to make up for the major flop that was Aduhlem. That drug, originally co-marketed between Biogen and Eisai, was approved in AD in 2021, but was a commercial flop and has been pushed to the side over questions on efficacy, safety and pricing. Leqembi is a much brighter prospect, as analysts at GlobalData see the drug as poised to realize $12.9 billion through 2028. Eisai has said it will take launch slow and steady and build up to its blockbuster sales over time.


Brenzavvy

Active ingredient: Bexagliflozin

Disease: Type 2 diabetes

Sales estimate: N/AApproved: Jan. 20

Company: TheracosBio      

The scoop: Approved to help improve glycemic control in adults with Type 2 diabetes, the 20 mg tablets of bexagliflozin are designed to be taken once a day alongside diet and exercise. The SGLT2 inhibitor helps lower blood sugar by preventing the kidneys from reabsorbing sugar created by the body, which then leaves through the urine. The drug class has become a fixture in treating Type 2 diabetes, with Brenzavvy going up against SGLT2 rivals like AstraZeneca’s Farxiga and Eli Lilly and Boehringer Ingelheim’s Jardiance. This summer, TheracosBio teamed up with Mark Cuban’s online distributor Cost Plus Drug Company to offer Brenzavvy at a price that significantly undercuts the competition, charging $47.85 plus shipping for a 30-day supply compared to $578 for a monthlong regimen of Jardiance or $551 for Farxiga. The drug is also the first oral therapy to make it to market for cats with diabetes mellitus.


Jaypirca

Active ingredient: PirtobrutinibDisease: Mantle cell lymphoma

Peak sales estimate: $1 billion

Approved: Jan. 27

Company: Eli Lilly

The scoop: Lilly joined a crowded marketplace with its accelerated approval of BTK inhibitor Jaypirca. By the time Lilly arrived, the FDA had already approved three other BTK inhibitors: AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa. But Lilly believes it has an ace up its sleeve as Jaypirca works differently, and Lilly is mainly angling its drug at a specific market. The differentiation comes from Jaypirca's mechanism of action: While its rivals can’t be used sequentially, Jaypirca is a non-covalent and thus binds to BTK differently and can be used after progression on another BTK inhibitor. The drug won its first speedy approval in mantle cell lymphoma, a rare type of B cell non-Hodgkin lymphoma (NHL), but in December Jaypirca grabbed a bigger label when the FDA gave it a speedy nod to treat chronic or small lymphocytic lymphoma in patients who have received at least two prior lines of therapy. 

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Orsurdu

Active ingredient: Elacestrant

Disease: Breast cancer

Peak sales estimate: $3 billion

Approved: Jan. 30

Company: Menarini

The scoop: The selective estrogen receptor degrader (SERD) is for postmenopausal women or adult men with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer who've experienced disease progression following at least one line of endocrine therapy. In this field, family-owned Menarini beat European giants to the finish line. In 2022, Roche and Sanofi flunked trials in breast cancer for their respective oral SERD candidates. Stemline—a subsidiary of Menarini—will commercialize the treatment in the U.S. Two months after Menarini acquired Stemline for $677 million in 2020, it bought commercial rights for elacestrant from Radius Health for $30 million plus $320 in milestone payments. Eisai was the original owner of the drug before selling off to Radius in 2006. The FDA has also blessed a companion diagnostic, the Guardant360 CDx assay, which helps identify patients eligible for Orserdu.


Jesduvroq

Active ingredient: Daprodustat

Disease: Anemia caused by chronic kidney disease

Sales estimate: $304 million (2028)

Approved: Feb. 1

Company: GSK

The scoop: When it came to getting an oral anemia drug over the FDA's finish line, the industry's third try in the hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) drug class was the charm. The coveted class-first approval means Jesduvroq can be used to treat patients with anemia caused by chronic kidney disease (CKD) who have been on dialysis for at least four months. Before GSK's approval, the FDA shot down similar applications from partnerships between Akebia/Otsuka and AstraZeneca/FibroGen. Despite Jesduvroq making HIF-PHI history, it seems only patients in the U.S. and Japan will benefit. A green light in Europe looked all but a done deal until GSK cited potential rivals and reduced market opportunities as reasons for withdrawing the EU application and abandoning efforts in other countries. Evaluate now estimates 2028 sales of $304 million compared to GSK’s original peak sales calculation of $600 million to $1.2 billion.


Lamzede

Active ingredient: Velmanase alfaDisease: Alpha-mannosidosis (AM)Sales estimate: N/AApproved: Feb. 16Company: Chiesi

The scoop: In a span of three months, Chiesi gained FDA approval for two of its enzyme replacement therapies. The second came in May when the U.S. regulator signed off on Fabry disease drug Elfabrio. But in February, Lamzede was approved as the first enzyme replacement therapy to treat children and adults with non-central nervous system manifestations of AM, a lysosomal storage disease. Those with the ultra-rare genetic condition—which affects one in every 500,000 to 1 million—lack the enzyme that breaks down sugars, causing accumulation in various tissues. Symptoms present in a variety of ways including impaired hearing, speech and cognitive function. Chiesi acquired the drug when it purchased Zymenex in 2013. Five years later, Italy-based and family-owned Chiesi gained approval for velmanase alfa in Europe. Lamzede is an intravenous powder priced at $4,221 per weekly injection, bringing its annual cost to $220,000.


Altuviiio

Active ingredient: Recombinant Fc-VWF-XTEN fusion protein

Disease: Hemophilia A

Sales estimate: $1.1 billion (2028)

Approved: Feb. 22

Companies: Sanofi and Sobi

The scoop: Altuviiio is Sanofi and Sobi’s attempt to reclaim the hemophilia A market after their established player Eloctate was knocked off course by the arrival of Roche’s blockbuster Hemlibra. The partners secured Altuviiio’s approval for routine prophylaxis and on-demand treatment to aid in bleeding episodes, as well as for surgery management for adults and children. But what set the factor VIII replacement therapy apart is its extended half-life, which Sanofi claimed is three to four times longer than rival options. So far, the drug hasn’t disappointed, with Sanofi execs praising the drug's €46 million ($50 million) in third-quarter sales as helping to offset generic competition to its multiple sclerosis drug Aubagio. With Japanese approval already in the bag and the EU currently considering a submitted application, it remains to be seen whether the treatment will follow in the blockbuster footsteps of its rival Hemlibra.


Filspari

Active ingredient: Sparsentan

Disease: Primary immunoglobin A nephropathy (IgAN)

Sales estimate:  $713 million (2028)

Approved: Feb. 27

Company: Travere Therapeutics

The scoop: The first-in-class treatment gained an accelerated approval for IgAN patients who are at risk of rapid disease progression. But the status of the approval is uncertain after a confirmatory trial came up short. Travere plans to apply for full approval in early 2024. Filspari is the first non-immunosuppressive therapy approved for IgAN. It works by blocking both endothelin and angiotensin receptors, which separates it from the angiotensin-targeting ACE inhibitors and ARBs—drugs that have been repurposed for IgAN because of their ability to lower blood pressure. Because the drug can do liver damage, the FDA has requested it be classified into a Risk Evaluation Mitigation Strategy (REMS) safety program. Travere is also developing Filspari for another kidney disorder, focal segmental glomerulosclerosis (FSGS). The company, formerly known at Retrophin, renamed itself to distance it from disgraced founder and financial criminal Martin Shkreli.


Skyclarys

Active ingredient: Omoveloxolone

Disease: Friedreich’s ataxia

Peak sales estimate: $740 million

Approved: Feb. 28

Company: Reata

The scoop: The FDA nod for Skyclarys was the first approval in the 21-year history of Reata. Then, five months later, Biogen bought out the Texas company for $7.3 billion in order to pair up Skyclarys with its established commercial infrastructure in neurology. Skyclarys is the first treatment for FA, a hereditary neurological disorder that strikes during adolescence and leaves patients wheelchair-bound and often cuts short their lives. It is an oral, once-daily Nrf2 transcription factor that binds to the Keap1 gene, restoring mitochondrial function and reducing inflammation. In 2020, the FDA turned thumbs down on the drug despite results from a study that showed the treatment led to significant improvement in walking at Week 48 over placebo. But regulators needed to see more over a longer timespan to sign off on it. The oral treatment is priced at $370,000 annually. Analysts with SVB have put the med's peak sales opportunity at $740 million.


Zavzpret

Active ingredient: Zavegepant

Disease: Migraine

Sales estimate: $649 million (2028)

Approved: March 9

Company: Pfizer

The scoop: The calcitonin gene-related peptide (CGRP) receptor antagonist has the same mechanism of action as Pfizer’s blockbuster-to-be migraine pill Nurtec. Both were gained in Pfizer’s $11.6 billion buyout of Biohaven in 2022. The key difference with Zavzpret: It is a nasal spray. While previous triptan treatments constrict blood vessels to reduce migraine pain, CGRPs work by blocking the receptors that cause inflammation. A spray can be an answer for patients who have nausea with their migraines and have difficulty keeping pills down. Study results showed that Zavypret can work as quickly as 15 minutes and its effects can last up to 48 hours. Pfizer launched Zavzpret in the third quarter at a cost of $1,168 for six sprays. It will face formidable competition, including from Eli Lilly's Emgality, Amgen's Aimovig, Teva's Ajovy and AbbVie's Qulipta, an oral drug approved last year. Another migraine nasal spray with a different mechanism of action is Trudhesa from Impel.


Daybue

Active ingredient: Trofinetide

Disease: Rett syndrome

Peak sales estimate: $650 million

Approved: March 10

Company: Acadia Pharmaceuticals

The scoop: Acadia Pharmaceutical’s Daybue is the first treatment approved for patients with the neurodevelopmental disorder Rett syndrome. The disease, which impacts an estimated 6,000 to 10,000 people in the U.S., limits patients' ability to communicate and leaves them “trapped in their own body,” Acadia’s head of rare diseases Kathie Bishop, Ph.D., said in an interview. During Daybue's first full quarter on the market, sales hit $66.9 million. J.P. Morgan Securities analysts have called the drug’s market performance “stunning” and project full-year 2023 revenues to reach $132 million, with the figure growing to $280 million in 2024. The analysts note that peak U.S. sales could come out to up to $650 million, FirstWord Pharma reports. Acadia licensed the med from Australia’s Neuron Pharmaceuticals in 2018 and expanded the agreement earlier this year to include rights outside of North America.


Zynyz

Active ingredient: Retifanlimab

Disease: Merkel cell carcinoma

Peak sales estimate: $41 million 

Approved: March 22

Company: Incyte

The scoop: After a 2021 rejection to treat squamous cell anal carcinoma, Incyte was able to turn around and score an FDA approval for PD-1 inhibitor retifanlimab last spring. In late March, the FDA signed off on the checkpoint inhibitor to treat metastatic or recurrent locally advanced Merkel cell carcinoma, a treatment field already occupied by Merck’s Keytruda and Merck KGaA’s Bavencio. In a twist, the FDA nod came as a surprise to industry watchers because Incyte hadn’t announced its application filing or a decision date before the official word came down, analysts with William Blair pointed out. At the time of the nod, the WB team said they saw a “limited opportunity” for the drug given the crowded checkpoint inhibitor field. For their part, analysts at Leerink projected a $41 million peak sales opportunity for the drug between the U.S. and Europe. 


Rezzayo

Active ingredient: Rezafungin

Disease: Candidiasis and candidemia infections

Peak sales estimate: $348 million

Approved: March 22

Companies: Cidara and Melinta

The scoop: This is the first approval in the 11-year history of San Diego-based Cidara Therapeutics. Rizzayo treats bloodstream infections in hospitalized patients. The next-generation echinocandin is the first new treatment option in the indication in more than a decade. In January, the FDA’s Antimicrobial Drugs Advisory Committee voted 14-1 to recommend it for approval. In a study, the drug showed non-inferiority to the standard-of-care treatment, Merck’s Cancidas, which now is available as a generic. With the nod, Cidara gained a $20 million milestone payment from Connecticut-based Melinta, which paid $30 million in 2022 for licensing rights to the drug in the U.S. In total, Cidara could reap $460 million, including milestones and tiered royalties. Mundipharma of the U.K. markets the drug outside of the U.S. Melinta launched Rezzayo in July at a wholesale acquisition cost of $1,950 for a single injection.


Joenja

Active ingredient: Leniolisib

Disease: Phosphoinositide 3-kinase delta syndrome (APDS)

Peak sales estimate: $200 million to $300 million

Approved: March 24

Company: Pharming Group

The scoop: With the March FDA nod, Joenja became the first disease-modifying treatment for the genetic condition, which was discovered just 10 years ago. Patients have been given antibiotics, immunosuppressants and immunoglobulin replacement drugs to deal with the symptoms. Roughly 500 people worldwide are being treated for APDS. Netherlands-based Pharming bought the drug from Novartis for $20 million in 2019, hoping to take advantage of its marketing infrastructure for its only other commercial product Ruconest, which treats a similar condition, hereditary angioedema (HAE). With the approval, Pharming made a $10.5 million milestone payment to Novartis. As commercial goals are reached, Novartis could receive up to $190 million more, plus tiered royalties. Pharming launched Joenja in April and has priced it at $750 per tablet, which is taken twice daily, bringing its annual price tag to $547,500.


Qalsody

Active ingredient: Tofersen

Disease: Amyotrophic lateral sclerosis (ALS)

Peak sales estimate: $300 million

Approved: April 25

Companies: Biogen and Ionis 

The scoop: It wouldn’t be a Biogen approval without a little controversy. Ionis-partnered amyotrophic lateral sclerosis (ALS) med tofersen was granted an accelerated approval for patients with a SOD1 gene mutation, to be sold as Qalsody. The FDA stopped short of a full approval, agreeing with an FDA advisory committee that had recommended against doing so due to a phase 3 failure. Nevertheless, SVB Securities analysts have estimated Qalsody could see peak sales of $300 million. Biogen CEO Chris Viehbacher heralded the drug as a major milestone for science, but a drug that may not be a huge revenue bringer for the company, during a third-quarter earnings call in November. The therapy is not yet included in Biogen’s sales breakout but lumped together in the “other” product revenue category that includes foundering Alzheimer’s drug Aduhelm. That segment brought in $3.7 million worldwide as of the end of the third quarter.  


Vowst

Active ingredient: Fecal microbiota

Disease: Clostridioides difficile infection

Sales estimate: $112 million (2024)

Approved: April 26

Companies: Seres Therapeutics and Nestlé Health Science 

The scoop: Odds are that somewhere along the line, you’ve seen an ad for a probiotic, a supplement consisting of a small microorganism meant to promote the good bacteria in your body. But in 2023, the FDA went a step further, approving Vowst as the first oral-fecal microbiota product. At its most crass, Vowst is a poop-based product, derived from donor fecal samples that when introduced into a patient are intended to restore gut health to fight C. diff infection. The approval marked a significant moment for the microbiome-based therapy field but also for Flagship Pioneering specifically, the venture creation firm that launched Seres Therapeutics. Prior attempts by Flagship had fallen flat, including the shuttering of fellow microbiome biotech Kaleido in April 2022. Vowst was also the second approved product to come from Flagship’s portfolio of companies after Moderna’s COVID vaccine success. 


Arexvy

Active ingredient: RSVPreF3

Disease: Respiratory syncytial virus (RSV)

Peak sales estimate: $3.6 billion

Approved: May 3

Company: GSK

The scoop: GSK’s Arexvy became the world’s first approved vaccine for RSV, creating a new prophylaxis against a virus that in many cases is no worse than a common cold. But in the very young and the elderly, RSV can turn into a serious infection and prove fatal. Hot on GSK’s heels however was Pfizer, which just several weeks later nabbed an approval for its rival RSV vaccine Abrysvo. Like Arexvy, Pfizer's shot also nabbed an older-adult label but then went on to get a green light as a maternal vaccine to prevent RSV in infants, something Arexvy doesn’t have. But the Pfizer shot also needs to compete with Sanofi and AstraZeneca’s antibody Beyfortus, which is given directly to infants. GSK is already on a strong launch trajectory, with Arexvy pulling in £709 million ($860 million) in the third quarter of 2023, more than doubling the $375 million haul from Pfizer’s shot during the same period. GSK is predicting around £3 billion in peak annual sales for its vaccine.


Elfabrio

Active ingredient: Pegunigalsidase alfa

Disease: Fabry disease

Sales estimate: N/A

Approved: May 9

Companies: Protalix and Chiesi

The scoop: In a span of three months, Chiesi gained FDA approval for two of its enzyme replacement therapies. Besides Elfabrio, the other approval came in February when the U.S. regulator signed off on Lamzede to treat alpha-mannosidosis. Protalix developed Elfabrio and family-owned Chiesi is commercializing it. Chiesi paid Protalix $20 million upon its FDA approval. Elfabrio was endorsed for adults with Fabry disease, a rare, genetic, progressive condition that causes an accumulation of fatty deposits in lysosomes and strikes roughly 1 in 50,000. The fats collect in blood vessels and tissues, raising the risk of heart attack, stroke and kidney failure. The FDA green light came four days after Europe granted a marketing license to the treatment. Elfabrio was fast-tracked for approval by the FDA in 2018 but pandemic-related inspection delays and a complete response letter slowed its roll. It is infused and is priced at $4,364 for a single-dose 10 mg vial.


Veozah

Active ingredient: Fezolinetant

Disease: Menopause-related vasomotor syndromes

Sales estimate: $2.2 billion (2025)

Approved: May 13

Company: Astellas

The scoop:  Vasomotor symptoms, or hot flashes and night sweats, make up some of the most dreaded and common characteristics of menopause. Veozah represents a new option as the first nonhormonal neurokinin 3 (NK3) receptor antagonist approved for the condition. The nonhormonal aspect of this drug is key for people who can’t take hormone therapies. The med works by blocking a chemical in the brain that regulates the body’s temperature and becomes unbalanced as estrogen declines during menopause. Veozah’s approval was delayed in February by a last-minute extended FDA review after Astellas had already used a priority-review voucher to take four months off of the timeline. The company expects sales to “grow rapidly” in the coming years, CEO Naoki Okamura said at the time of the approval. Astellas expects full-year 2023 Veozah sales to reach JPY 49.3 billion ($340 million) and JPY 300 billion ($2 billion) by 2025, Bloomberg reports. 


Miebo

Active ingredient: Perfluorohexyloctane

Disease: Dry eye disease symptoms

Peak sales estimate: $1 billion

Approved: May 18

Companies: Bausch + Lomb and Novaliq

The scoop: Joining a crowded dry eye disease market, Bausch + Lomb and Novaliq’s Miebo is the first treatment to directly target tear evaporation, targeting what Bausch + Lomb CEO Brent Saunders has called a “significant unmet need.” Dry eye disease impacts millions of people in the U.S. and is one of the most common ocular surface disorders. In two phase 3 trials testing Miebo in a total of 1,217 patients over 57 days, patients experienced lasting symptom relief starting as early as day 15. The med, which marked Bausch + Lomb’s first approval since closing on its IPO ahead of an upcoming separation from Bausch Health, launched over the third quarter. Analysts predict Miebo will generate peak-year sales exceeding $1 billion, according to a report from CarelonRx.


Vyjuvek

Active ingredient: Beremagene geperpavec-svdt

Disease: Dystrophic epidermolysis bullosa

Sales estimate: $273 million (2024)

Approved: May 19

Company: Krystal Biotech

The scoop: Krystal Biotech’s Vyjuvek is not only the first treatment for the rare and devastating skin disease dystrophic epidermolysis bullosa (DEB), but it's also the first-ever topical gene therapy approved by the FDA. The medicine comes in a gel form that DEB patients can use at home to heal up their DEB wounds, which are caused by mutations in the COL7A1 gene that lead the skin's layers to separate easily. Analysts at Evercore ISI have recently raised their Vyjuvek sales forecast to $273 million in 2024. Krystal finished the third quarter of last year with 284 start forms for the treatment; it expects an 85% conversion rate to prescriptions. The drug brought in third-quarter sales of $8.6 million. Next up is a potential European approval, which the company is anticipating in the second half of 2024. It will also file for approval in Japan in early 2024.


Epkinly

Active ingredient: Epcoritamab

Disease: Diffuse large B-cell lymphoma and high-grade B-cell lymphoma

Peak sales estimate: $2.75 billion

Approved: May 19

Companies: AbbVie and Genmab

The scoop: After an approval in May, Epkinly is going toe-to-toe with Roche’s Columvi in large B-cell lymphoma. Epkinly not only arrived about a month earlier than its rival, but it also boasts a broader label. Compared with Columvi, Epkinly’s label also includes high-grade B-cell lymphoma. By a cross-trial comparison, Epkinly’s 61% overall response rate in a group of patients who had tried a median of three prior lines of therapy looked better than Columvi’s 51.6% result, although Columvi showed a longer duration of response. Epkinly also appeared to have a better safety profile, boasting a lower rate of cytokine release syndrome. What’s more, Epkinly holds a convenience edge as a subcutaneous formulation, although Roche is trying to change Columvi’s intravenous infusion route. Epkinly is given indefinitely, whereas Columvi comes with a fixed duration. Jefferies analysts previously predicted Epkinly could reach about $2.75 billion in peak sales.


Xacduro

Active ingredients: Sulbactam and durlobactam

Disease: Acinetobacter pneumonia

Sales estimate: $82 million (2028)

Approved: May 23

Company: Innoviva

The scoop: Xacduro targets bacteria known as Acinetobacter baumannii, a pathogen that can cause infections in the blood, urinary tract and lungs. The treatment is for hospital-acquired and ventilator-associated bacterial pneumonia. Acinetobacter has drug-resistant qualities and has been identified as a threat by the WHO and the CDC. It is primarily spread through person-to-person contact or direct contact with contaminated surfaces. In the U.S., more than 8,500 Acinetobacter infections are reported annually. The nod came after an FDA advisory committee voted 12-0 to recommend approval. The co-packaged treatment is a combination of sulbactam, a beta lactam antibiotic, and durlobactam, a broad-spectrum beta lactamase inhibitor that tamps down Acinetobacter’s resistance, allowing the antibiotic to take effect. The treatment was co-developed by Zai Lab and AstraZeneca spinout Entasis Therapeutics, which was sold to Innoviva for $113 million.


Paxlovid

Active ingredients: Nirmatrelvir and ritonavir

Disease: COVID-19

Sales estimate: $3 billion (2024)

Approved: May 25

Company: Pfizer

The scoop: While not the first drug to treat COVID-19—that designation went to a repurposed Ebola med from Gilead—Pfizer did gain approval for the first COVID-specific treatment in Paxlovid, getting an emergency FDA authorization in December 2021 and a full approval in May 2023. Paxlovid sales initially skyrocketed for the Big Pharma as the drug brought in a massive $19 billion in 2022, making it one of the biggest-selling drugs of all time with the straightest launch trajectory for a megablockbuster ever seen. But as swiftly as the drug took off, it has since come crashing back down to Earth. In the third quarter of 2023, Paxlovid brought in just $202 million, echoing a similar revenue fall for Pfizer’s Comirnaty COVID vaccine. This comes as the emergency stage of the pandemic has drifted into an endemic one, leading to a major drop-off in demand for COVID shots and drugs.


Inpefa

Active ingredient: Sotagliflozin

Disease: Heart failure

Sales estimate: $333 million (2028)

Approved: May 26

Company: Lexicon

The scoop: It was a long road to approval for Inpefa, which was rejected by the FDA twice as a treatment for type 1 diabetes. But last year, the med was blessed to reduce the risk of heart failure in adults who have had the condition or have type 2 diabetes, chronic kidney disease and other cardiovascular risk factors. The drug has shown tantalizing potential to block both SLGT2 in the kidneys and SLGT1 in the intestines. With the nod, it became the only SGLT1/SGLT2 treatment on the market. The drug has been approved to treat type 1 diabetes in Europe, where it is known as Zynquista, but is not available there as license holder Guidehouse Germany has withdrawn (PDF) its marketing authorization. In 2015, the potential of sotagliflozin was recognized by Sanofi, which ponied up $300 million and committed $1.4 billion in possible milestone payments. When the first FDA rejection came, however, the French powerhouse paid Lexicon $260 million to exit the partnership.


Abrysvo

Active ingredient: RSVPreF2

Disease: Respiratory syncytial virus

Peak sales estimate: $2 billion

Approved: May 31

Company: Pfizer

The scoop: Abrysvo gained two approvals in 2023, the first for patients age 60 and older and the second for women who have been pregnant for 32 to 36 weeks to protect their soon-to-be-born babies. The second nod came a month after Sanofi and AstraZeneca secured FDA approval for their RSV immunization for infants up to 24 months old. RSV is the leading cause of hospitalization for infants and kills roughly 300 children younger than age 5 annually in the U.S. An FDA advisory panel ruled that evidence supported vaccination in the second and third trimesters of pregnancy but the regulator narrowed the window of use. The first FDA approval for Abrysvo came a month after GSK won endorsement of its shot Arexvy for seniors. A trial showed that Abrysvo achieved 67% protection against RSV-associated lower respiratory tract illness. Against more severe illness, the vaccine's efficacy came in at 86%. In the third quarter, Pfizer reported $375 million in sales of Abrysvo.


Columvi

Active ingredient: Glofitamab

Disease: Diffuse large B-cell lymphoma

Peak sales estimate: $2 billion

Approved: June 15

Company: Roche

The scoop: Columvi is Roche’s second CD20xCD3 T-cell engager, following Lunsumio, a follicular lymphoma treatment. The FDA's approval for Columvi followed AbbVie and Genmab’s rival diffuse large B-cell lymphoma (DLBCL) bispecific Epkinly. Jefferies analysts previously predicted Columvi could reach $2 billion in peak sales. For its two agents, Roche hopes a fixed-duration regimen can improve patients’ quality of life by allowing treatment-free time and potentially reducing some long-term side effects. Roche is also investigating the two bispecific antibodies in subcutaneous formulations, trying to play catch-up with Epkinly. In a recent phase 2 trial update, 55% of heavily pretreated patients who had achieved a complete response on Columvi remained in remission after two years. But the real battle is in early treatment settings. Roche in September started the phase 3 SKYGLO trial combining Columvi with the Polivy-R-CHP regimen in previously untreated DLBCL.


Elevidys

Active ingredient: Delandistrogene moxeparvovec

Disease: Duchenne muscular dystrophy (DMD)

Sales estimate: $1 billion

Approved: June 22

Company: Sarepta Therapeutics

The scoop: Sarepta’s DMD gene therapy scored accelerated approval in a restricted population of ambulatory patients after a rocky six-year development period plagued by safety concerns. Following a narrow advisory committee vote, the FDA cleared the med for treatment in patients ages 4 to 5. But Sarepta isn’t stopping there. It plans to file for a label expansion that would include “all DMD patients” and convert the nod into a full approval. That expansion would be supported by a phase 3 study that missed its primary endpoint. Still, Sarepta’s CEO Doug Ingram said that the study “met the standard” to prove evidence of effectiveness. The $3.2 million one-time gene therapy raked in $69 million in third-quarter sales amid what Ingram described as “a superb launch.” After the trial failed, analysts at RBC estimated the med can deliver $1 billion in annual sales and a peak of $3.4 billion if the FDA does expand the label, Investors Business Daily reported.


Litfulo

Active ingredient: Ritlecitinib

Disease: Alopecia areata

Sales estimate: $1 billion

Approved: June 23

Company: Pfizer

The scoop: In the alopecia areata arena, Eli Lilly’s Olumiant had a year-long head start after the JAK inhibitor’s 2022 approval, which was the first in this disease. But last summer, Pfizer crossed the FDA’s finish line with Litfulo, previously known as ritlecitinib. Pfizer’s JAK inhibitor is approved to treat adults—like the Eli Lilly treatment option—but it also boasts an approval to treat children 12 and older. Alopecia areata causes the body to attack its own hair follicles, which leads the patient's hair to fall out. The condition affects nearly 7 million people in the U.S. and roughly 147 million people around the globe, according to Pfizer. Pfizer has previously said the drug could generate more than $1 billion in the disease. It’s one of several new drugs Pfizer hopes can help grow its business amid a speedy decline in sales of COVID-19-related products. 


Rystiggo

Active ingredient: Rozanolixizumab-noli

Disease: Generalized myasthenia gravis (gMG)

Sales estimate: $990 million (2031)

Approved: June 26

Company: UCB

The scoop: Four months after Rystiggo’s approval, UCB pushed another gMG drug called Zilbrysq across the FDA finish line. While the peptide Zilbrysq is a targeted C5 inhibitor, Rystiggo is a subcutaneously infused monoclonal antibody that targets the neonatal Fc receptor. It is the first therapy approved to treat the two primary subsets of the gMG—those who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. UCB charges a list price of $6,050 per vial, with the dosage dependent on a patient's weight and administered depending on the prevalence of symptoms. The drug remains under review in Japan and Europe. There is formidable competition in the gMG space as AstraZeneca won approval for Ultomiris in the indication in 2022. More recently, Argenx gained an FDA nod for its own subcutaneous drug, Vyvgart Hytrulo. 


Ngenla

Active ingredient: Somatrogon-ghla

Disease: Growth hormone deficiency (GHD)

Sales estimate: $278 million

Approved: June 27

Company: Pfizer and OPKO Health

The scoop: The approval for Ngenla covers those ages 3 and older who have stunted growth because of inadequate secretion of endogenous growth hormone.  A decision on the next generation, once-weekly injected drug was initially set for October 2021, but the FDA asked Pfizer and OPKO for longer-term data, then in January 2022 the regulator sent a complete response letter to the companies. Without treatment, children will grow to a very short height in adulthood and puberty can be delayed. Ngenla’s thumbs up was supported by a phase 3 study, which evaluated its safety and effectiveness versus Genotropin, Pfizer’s standard-of-care drug that is administered daily. Compared with Genotropin, Ngenla achieved non-inferiority as measured by height increases after a year. Pfizer partnered with Miami-based OPKO on the therapy in 2014, paying $295 million up front. Ngenla sells for a list price of $8,300 monthly for a child weighing 75 pounds.


Lantidra

Active ingredient: Donislecel

Disease: Type 1 diabetes

Sales estimate: $200 million (2029)

Approved: June 28

Company: CellTrans

The scoop: Lantidra is the first FDA-approved cell therapy for Type 1 diabetes. Harvested from donated pancreas tissue, its islet beta cells are delivered into the livers of adult patients who still suffer dramatic swings in their blood sugar despite intensive insulin regimens. In clinical studies of 30 participants who received between one and three infusions, 21 patients did not need to take any extra insulin for at least a year—including 11 who went insulin-free for more than five years. Chicago-based startup CellTrans took gold in this field over long-watched companies such as Vertex and Sernova. Meanwhile, a day after the FDA trumpeted CellTrans’ green light, Eli Lilly announced it signed a deal to acquire Sigilon for a bargain price of $35 million. But the race continues as Lantidra’s liver infusions and its need for immunosuppressive drugs saw the majority of clinical trial participants have at least one serious adverse reaction.


Roctavian

Active ingredient: Valoctocogene roxaparvovec-rvox 

Disease: Hemophilia A 

Peak sales estimate: $2.2 billion 

Approved: June 29 

Company: BioMarin Pharmaceutical 

The scoop: BioMarin had to fight tooth and nail to get Roctavian past the FDA finish line, weathering both a rejection in 2020 and a review delay in early 2023. That approval came several months after CSL Behring’s hemophilia B gene therapy, Hemgenix, won approval to become the world’s most expensive drug at $3.5 million. Roctavian, for its part, carries a wholesale acquisition cost of $2.9 million. The drug also features an outcomes-based warranty program, which promises to reimburse government and commercial payers up to the full cost if it doesn’t live up to treatment expectations. Of the estimated 6,500 adults with severe hemophilia A in the U.S., BioMarin figures around 2,500 will be eligible to receive Roctavian. Unlike most gene therapies, BioMarin’s drug is approved to address a disease for which there are already many treatment options. That means Roctavian’s initial sales ramp will likely be slow, SVB Securities analysts have said.


Beyfortus

Active ingredient: Nirsevimab-alip 

Disease: Respiratory syncytial virus (RSV)

Peak sales estimate: $3 billion

Approved: July 17 

Company: Sanofi and AstraZeneca 

The scoop: This summer proved momentous for the RSV immunization field, with new vaccines coming to the fore from Pfizer and GSK. But before those shots won approval, Beyfortus snagged a world-first approval in Europe to protect against the seasonal disease in infants in November 2022. Fast-forward to July, when Sanofi and AZ’s drug passed muster with the FDA. The antibody will be critical to Sanofi’s plan to earn more than 10 billion euros in yearly immunization revenues by 2030. But despite big expectations for Beyfortus, supply has proved a limiting factor early in the drug’s U.S. launch. In October, the CDC recommended doctors prioritize available Beyfortus 100 mg doses for infants at the highest risk of severe RSV disease. At the time, Sanofi attributed the shortfall to “higher than anticipated demand,” which has affected supplies of the drug despite an “aggressive supply plan built to outperform past pediatric vaccine launches.” 


Cyfendus

Active ingredient: Anthrax vaccine adsorbed, adjuvanted

Disease: Post-exposure prophylaxis for Bacillus anthracis

Sales estimate: N/A

Approved: July 20

Company: Emergent BioSolutions

The scoop: Fifteen years after first partnering with BARDA to develop a new anthrax countermeasure—and four years after first supplying it to the U.S. government—Emergent claimed an FDA approval this year for its two-dose vaccine designed to be given after a confirmed exposure to the deadly pathogen. While anthrax spores can be found naturally in soil and animals, the pathogen has long posed a threat as a biological weapon. Under a prior pre-emergency use authorization, Emergent had been delivering millions of doses of the vaccine to the U.S. national stockpile. Cyfendus aims to be a next-generation, adjuvanted upgrade to its predecessor BioThrax, also developed by Emergent, which has been in use since the 1970s. In late November, BARDA exercised a $75 million option to purchase more doses of Cyfendus as it replaces its BioThrax reserves. Emergent also produces vaccines for smallpox and Ebola.


Vanflyta

Active ingredient: Quizartinib

Disease: Acute myeloid leukemia

Sales estimate: $313 million (2029)

Approved: July 20

Company: Daiichi Sankyo

The scoop: Lately, Daiichi has been riding the commercial success of its AstraZeneca-partnered cancer medicine Enhertu. But now the company has a new oncology offering to add to the mix in Vanflyta, an acute myeloid leukemia med that gained its FDA endorsement in July. Formerly known as quizartinib, the drug was previously rejected by the FDA in 2019. But thanks to the company’s persistence and the approval last year, Daiichi can now market its medicine against rival AML offerings from Novartis and Astellas. Daiichi acquired the therapy, its third oncology offering to gain a U.S. nod, back in 2014 when it acquired San Diego’s Ambit Biosciences for $410 million. Analysts with GlobalData have projected the drug’s sales will reach $313 million by 2029. 


Balfaxar

Active ingredient: Prothrombin complex concentrate

Condition: Pre-op blood thinner reversal

Sales estimate: N/A

Approved: July 21

Company: Octapharma

The scoop: It can be difficult to balance a patient’s blood. It needs to be thin enough to not form dangerous, stroke-causing clots, but not so thin that it puts them at a high risk of fatal bleeds. Balfaxar’s 2023 approval offered clinicians another way to put their thumb on the scale. Sold as a powder made from human plasma and given via IV, Balfaxar is designed to offer a quick way to reverse the effects of a class of long-term blood thinner medications in adult patients who are facing an urgently needed surgery or invasive procedure. Sold as Octaplex in Europe and Canada, it provides four clotting factors, as well as antithrombotic proteins, and aims to have a patient ready to go under the knife in about 30 minutes. Octapharma is also studying Balfaxar’s effects in patients taking direct oral anticoagulants such as Xarelto and Eliquis.


Xdemvy

Active ingredient: Lotilaner

Disease: Demodex blepharitis

Sales estimate: $652 million (2029)

Approved: July 25

Company: Tarsus Pharmaceuticals

The scoop: Some 25 million people in the U.S. with the eyelid disease Demodex blepharitis now have a treatment for their condition after the FDA’s July approval of Tarsus Pharmaceuticals’ Xdemvy. The drug targets Demodex mites, an ectoparasite infestation that can lead to inflammation in the skin, eyelashes, lash follicles, and nearby areas. Before Xdemvy, the roughly 1 in every 12 U.S. adults who suffered from the condition turned to tea tree oil and lid wipes, which don’t target the root cause of the disease. Tarsus’ drug is an eye drop formulation of pet flea and tick treatment lotilaner. In two trials involving more than 833 patients, investigators noted improvement in collarettes, which are areas of accumulation of the mite’s waste and eggs. In a corporate presentation (PDF), Tarsus has said the drug could become a blockbuster.  


Zurzuvae

Active ingredient: Zuranolone

Disease: Postpartum depression

Sales estimate: $200 million (2030)

Approved: Aug. 4

Companies: Biogen and Sage Therapeutics

The scoop: While drugmakers often celebrate new drug approvals, the FDA’s August nod for Biogen and Sage Therapeutics’ Zurzuvae was overshadowed by a larger rejection for the medicine. Specifically, the FDA approved the drug to treat postpartum depression, but it spurned the partners’ application for the treatment of major depressive disorder. At the time of the approval, Mizuho Securities analysts said the MDD nod would have been a “meaningfully larger opportunity” for Zurzuvae, with potential 2030 sales in that indication reaching $1.3 billion. In PPD, the drug can pull down around $200 million by 2030, the team said. Sage’s stock plummeted on the heels of the FDA decision, and the company subsequently laid off 40% of its staff to try to turn a profit from the limited FDA nod. 


Izervay

Active ingredient: Avacincaptad pegol intravitreal solution

Disease: Geographic atrophy (GA)

Peak sales estimate: $2.4 billion

Approved: Aug. 4

Company: Astellas

The scoop: Less than four months after Astellas bought out Iveric Bio for $5.9 billion, the Japanese company secured its FDA nod for the prime asset acquired in the acquisition, Izervay. Astellas made the purchase in hopes that Izervay can take some of the sting out of Xtandi's approaching loss of exclusivity. The approval came six months after Apellis became the first to get a GA treatment across the FDA finish line, scoring with Syfovre. In July, Syfovre lost some of its first-to-market advantage when it was flagged for rare cases of severe inflammation. Astellas charges $2,100 for a monthly dose of Izervay. The C5 inhibitor was approved after two phase 3 trials showed it provided a significant reduction in GA progression after 12 months. Izervay’s label includes warnings about endophthalmitis (inflammation), infections and intraocular pressure.


Talvey

Active ingredient: Talquetamab

Disease: Multiple myeloma

Peak sales estimate: $5 billion

Approved: Aug. 9

Company: Johnson & Johnson

The scoop: In August, Talvey became the first GPRC5D-targeted therapy to cross the FDA finish line and the company’s second bispecific antibody to win approval for multiple myeloma. The drug is approved as a fifth-line therapy, which is the same line as J&J’s Legend Biotech-partnered BCMA CAR-T Carvykti and bispecific Tecvayli. Talvey has shown anticancer activity in patients pretreated with a BCMA CAR-T, but the approval doesn’t restrict the new drug's use behind a BCMA agent. In a single-arm trial, Talvey induced a response in 72% of 32 patients who had previously tried a CAR-T therapy or a bispecific antibody. The drug’s overall response rate was similar in patients who had not received any BCMA agents. During a business update in December, J&J listed Carvykti, Tecvayli and Talvey as each having the potential to reach more than $5 billion in peak sales. Other companies developing GPRC5D therapies include Bristol Myers Squibb and Roche.


Elrexfio

Active ingredient: Elranatamab

Disease: Multiple myeloma

Sales estimate: $536 million (2029)

Approved: Aug. 14

Company: Pfizer

The scoop: With Elrexfio, Pfizer entered a crowded BCMA market for multiple myeloma. The off-the-shelf bispecific antibody competes directly with Johnson & Johnson’s Tecvayli, but other rivals include CAR-T therapies from Bristol Myers Squibb and the partnership between J&J and Legend Biotech. A June update of the phase 2 MagnetisMM-3 study showed Elrexfio shrank tumors in 61% of a group of heavily pretreated patients, including 35% who achieved complete responses or better. Elrexfio’s tumor response data weren’t so different from Tecvayli’s, and Pfizer arrived only less than a year after J&J. But J&J’s humongous, multi-faceted multiple myeloma portfolio will be difficult for others to match. GlobalData previously predicted Elrexfio could reach $536 million in sales by the end of 2029 whereas Pfizer has guided to $4 billion in peak sales.


Sohonos

Active ingredient: Palovarotene

Disease: Fibrodysplasia ossificans progressiva  

Peak sales estimate: N/A

Approved: Aug. 16 

Company: Ipsen Biopharmaceuticals

The scoop: Back in 2019 Ipsen forked over $1 billion to acquire Clementia Pharmaceuticals for its rare disease drug palovarotene. At the time, Ipsen referred to the candidate as “largely de-risked.” But it would be another four years—marred by two clinical trial holds, a complete response letter, a European Commission rejection and more—before the drug finally crossed the FDA’s finish line. Now christened Sohonos, the retinoic acid receptor gamma agonist is cleared to treat the ultra-rare bone disease fibrodysplasia ossificans progressive (FOP) in females ages 8 and older and males ages 10 and older. The disease, which affects roughly 400 people in the U.S., leads to loss of mobility, forcing most patients to use a wheelchair by age 30 and cutting their life expectancy to around 56 years. Roche developed palovarotene but sold its worldwide rights to Canadian company Clementia in 2013 after the treatment flunked a phase 2 trial in emphysema.


Veopoz

Active ingredient: Pozelimab

Disease: CD55-deficient protein-losing enteropathy (CHAPLE disease)

Sales estimate: N/A

Approved: Aug. 18

Company: Regeneron

The scoop: With fewer than 10 CHAPLE disease patients in the U.S. and less than 100 worldwide, Regeneron’s lgG4 antibody Veopoz represents a welcome treatment option for the ultra-rare, often fatal hereditary immune disease. The med marks the first approved therapy for the condition, which is characterized by an overactive complement system that can attack the body’s own cells. In a phase 2/3 study testing the drug intravenously in 10 patients ages 3 to 19, all 10 maintained normal serum albumin and serum lgG levels by week 12 and maintained those levels through at least 48 weeks. Veopoz is also being assessed as a combination therapy with Alnylam’s siRNAi C5 inhibitor cemdisiran to treat other complement-mediated disorders, such as the blood disease paroxysmal nocturnal hemoglobinuria and neuromuscular condition myasthenia gravis. Regeneron priced the drug at $34,615 per single-use vial and launched it during 2023’s third quarter.


Aphexda

Active ingredient: Motixafortide

Use: To mobilize hematopoietic stem cells in patients with multiple myeloma

Peak sales estimate: $360 million

Approved: Sept. 8

Company: BioLineRx

The scoop: Two decades after its founding, Israel’s BioLineRx nabbed its first commercial approval in September for Aphexda. The FDA approved the drug in combo with filgrastim to mobilize hematopoietic stem cells to the peripheral blood for collection and autologous stem cell transplantation (ACST) in patients with multiple myeloma. At the time of the nod, BioLineRx said Aphexda was the first innovation in stem cell mobilization for multiple myeloma in a decade. Founded in 2003 with financial backing from Israeli drugmaker Teva Pharmaceutical Industries, BioLineRx is also developing therapies against sickle cell disease, pancreatic cancer, and other solid tumors. After the Aphexda approval, BioLineRx said its research suggests the U.S. market for mobilization agents used in stem cell transplants is around $360 million annually. 


Ojjaara

Active ingredient: Momelotinib

Disease: Myelofibrosis

Peak sales estimate: $627 million

Approved: Sept. 15

Company: GSK

The scoop: Ojjaara represents a new challenger to Incyte’s well-established Jakafi. GSK's JAK inhibitor won the nod to treat intermediate- or high-risk myelofibrosis in patients with anemia. The approval covers patients regardless of their treatment history as long as they are anemic. The broader-than-expected label makes GSK believe Ojjaara could “comfortably” exceed $1 billion in sales, GSK CCO Luke Miels said, though Morningstar pegs the med's peak sales opportunity at $627 million. Previously, Ojjaara failed to outperform Jakafi on a myelofibrosis symptom scorecard in a phase 3 trial in JAK-naïve patients. The drug’s label shows that Ojjaara showed a numerically lower spleen volume response rate compared with Jakafi in anemic patients in that trial. But GSK believes the drug could compete in anemic patients because Jakafi might worsen anemia in some patients. GSK obtained Ojjaara through its $1.9 billion buyout of Sierra Oncology in 2022.


Exxua

Active ingredient: Gepirone

Disease: Major depressive disorder

Peak sales estimate: $1 billion

Approved: Sept. 22

Company: Fabre-Kramer Pharmaceuticals

The scoop: When the FDA approved Fabre-Kramer Pharmaceuticals’ major depressive disorder drug Exxua back in the fall, the regulatory milestone was decades in the making. Invented in 1986 by Bristol Myers Squibb, the drug changed hands several times before the eventual FDA nod. Between two periods of ownership by Fabre-Kramer, Organon stumbled at the FDA twice. Afterward, Fabre-Kramer linked up with GSK in a deal that did not pan out. Despite the three FDA rejections for the med, also known as gepirone, Fabre-Kramer pushed through and eventually convinced the FDA of the drug’s efficacy in MDD. Exxua is the first MDD medicine that selectively targets the serotonin 1A receptor, which is key in regulating mood and emotion, according to the drugmaker. At the time of the approval, Fabre-Kramer said it expected to launch the drug in early 2024. The company has placed a potential sales target of $1 billion on the medicine.


Pombiliti

Active ingredient: Cipaglucosidase alfa-atga and miglustat  

Disease: Pompe disease 

Peak sales estimate: $1.2 billion 

Approved: Sept. 28 

Company: Amicus Therapeutics 

The scoop: Look out Sanofi: With U.S. approval for its combination therapy Pombiliti and Opfolda, Amicus is gunning for the Pompe disease crown. Originally slated for an FDA decision in October 2022, Amicus’ initial bid was foiled by COVID-related travel restrictions. But following a European nod in March, Pombiliti and Opfolda secured their U.S. green light. The endorsement tees up the combo therapy for adults living with late-onset Pompe disease who aren’t improving on their current enzyme replacement therapy (ERT). Pombiliti is an infused long-term ERT while Opfolda is an oral stabilizer, which reduces the loss of enzyme activity in the blood during infusion. Amicus believes the Pompe disease market could hit $1.8 billion by 2027. Upon scoring approval, Amicus said it was set to launch imminently in the U.S., where Pombiliti and Opfolda are priced at $650,000 annually for a patient of median weight. 


Rivfloza

Active ingredient: Nedosiran 

Disease: Primary hyperoxaluria type 1  

Sales estimate: N/A 

Approved: Oct. 2 

Company: Novo Nordisk 

The scoop: While much of the attention on Novo Nordisk revolves around the company’s skyrocketing diabetes and obesity franchises, the Danish drugmaker hasn’t lost sight of smaller indications. Look no further than Rivfloza, a monthly, subcutaneous RNAi therapy that won U.S. approval to treat primary hyperoxaluria type 1 (PH1) in kids over 9 years old and in older adults with relatively preserved kidney function. Novo’s drug is the second FDA-approved therapy for PH1, following Alnylam’s rival ribonucleic acid interference drug Oxlumo. Novo plans to launch in early 2024. Novo estimates that there are about 2,000 patients living with PH1 in the U.S., many of whom are thought to be undiagnosed. Before Novo entered the scene, nedosiran was Dicerna’s lead pipeline candidate. Novo picked up the med when it purchased Dicerna for $3.3 billion in 2021. As for Alnylam’s rival drug, Oxlumo generated $29 million in 2023’s third quarter.


Velsipity

Active ingredient: Etrasimod

Disease: Ulcerative colitis

Sales estimate: $1 billion (2031)

Approved: Oct. 12

Company: Pfizer

The scoop: Since May 2021, Bristol Myers Squibb’s Zeposia has been the lone S1P modulator approved to treat ulcerative colitis in the United States. But Pfizer changed that with its October 2023 approval for Velsipity, an oral, once-daily treatment for adults with moderate to severe active UC. The drug carries big commercial expectations at Pfizer, given that the drugmaker ponied up $6.7 billion for the medicine’s former owner, Arena Pharmaceuticals. Pfizer inked that deal in late 2021 and completed the acquisition the following spring. Velsipity represents an emerging threat to BMS’ Zeposia, while Pfizer itself bills the new entrant as a potential best-in-class option. Last year, Leerink Partners estimated Velsipity’s UC sales could reach $1 billion by 2031. After the UC nods, both companies hope to expand their S1P drugs to other autoimmune diseases, including the other form of inflammatory bowel disease, Crohn’s disease.


Zilbrysq

Active ingredient: Zilucoplan

Disease: Generalized myasthenia gravis (gMG)

Peak sales: N/A

Approved: Oct. 17

Company: UCB

The scoop: Within hours, Brussels-based UCB scored FDA green lights for Zilrysq and plaque psoriasis treatment Bimzelx. Additionally, the endorsement for Zilbrysq is UCB’s second in the indication this year, coming on top of the FDA signing off on Rystiggo in June. While the peptide Zilbrysq is a targeted C5 inhibitor, Rystiggo is a subcutaneously infused monoclonal antibody that targets the neonatal Fc receptor. The thumbs-up for Zilbrysq is for adults with gMG who are anti-acetylcholine receptor (AChR) antibody-positive. The medicine becomes the first once-daily, subcutaneous gMG-targeted therapy that can be self-administered. UCB acquired the drug in a $2.5 billion buyout of Ra Pharmaceuticals in 2019. There is formidable competition in the gMG space. Last year, AstraZeneca won approval for Ultomiris. In June, Argenx gained an FDA nod for Vyvgart Hytrulo. Zilrysq was approved in Europe in December.


Bimzelx

Active ingredient: Bimekizumab

Disease: Plaque psoriasis

Peak sales: $4.3 billion

Approved: Oct. 17

Company: UCB

The scoop: After overcoming manufacturing issues that resulted in an initial rejection, UCB’s psoriasis med Bimzelx finally nabbed approval in October and became available to patients in November. The company had been waiting on an FDA decision since 2021 when COVID-related travel restrictions held up manufacturing inspections. But the long delay gave UCB time to collect data that could support further indications and allow the company to “move forward rapidly” with additional filings, UCB has said. CEO Jean-Christophe Tellier has touted Bimzelx’ “best-in-disease” profile in both psoriasis and psoriatic arthritis, noting that the drug offers a “tremendous value proposition.” In trials, 85% to 91% of treated patients saw clear or almost clear skin by week 16. The company is also looking to go after FDA approvals in generalized pustular psoriasis and non-radiographic axial spondylarthritis to match overseas nods in both indications.


Agamree

Active ingredient: Vamorolone 

Disease: Duchenne muscular dystrophy (DMD)

Sales estimate: $158 million over 5 years  

Approved: Oct. 26 

Company: Santhera and Catalyst Pharmaceuticals  

The scoop: In many ways, Swiss drugmaker Santhera sees its recent approval in DMD as a launchpad for other future Agamree indications. The company is now anxious to see how many disorders the first-in-class corticosteroid can treat. Agamree, also known as vamorolone, is what’s known as a dissociative steroid. The idea behind this class of drug is to retain the properties that give steroids their efficacy while dodging unpleasant side effects. In turn, Agamree could help tackle a range of inflammatory conditions whose treatments leave patients overwhelmed with side effects. Among the conditions vamorolone could potentially treat are asthma, inflammatory bowel disease, rheumatoid arthritis and multiple sclerosis. As it develops vamorolone, Santhera will focus on rare diseases first. Santhera is forecasting the drug's sales potential in Europe to exceed 150 million euros ($158 million) in five years, the company’s CEO Dario Eklund said in a recent interview.


Omvoh

Active ingredient: Mirikizumab

Disease: Ulcerative colitis

Sales estimate: $1.2 billion (2028)

Approved: Oct. 26

Company: Eli Lilly

The scoop: Eli Lilly had trouble getting several of its new drug applications over the finish line in 2023. But the company was able to win a nod for Omvoh, its new ulcerative colitis entrant. Rejected by the agency in April 2023 over manufacturing problems, the drug in October 2023 became the first IL-23 inhibitor to enter the UC arena. Meanwhile, biologics from AbbVie, Johnson & Johnson and Takeda were already vying for market share before Omvoh’s approval, so Lilly will try to flex its marketing muscle to help its late-comer gain traction. Besides the UC use, Lilly’s drug previously beat placebo on both clinical remission and endoscopic response in patients with Crohn’s disease. Omvoh is one of four key launches that Lilly expects will drive its growth in the coming years. Aside from the April rejection for Omvoh (mirikizumab), the FDA also turned away Lilly’s applications for Alzheimer’s prospect donanemab and atopic dermatitis candidate lebrikizumab last year. 


Loqtorzi

Active ingredient: Toripalimab

Disease: Nasopharyngeal carcinoma

Peak sales estimate: $200 million

Approved: Oct. 27

Companies: Coherus BioSciences and Junshi Biosciences

The scoop: Coherus BioSciences and Junshi Biosciences' Loqtorzi is the first China-made PD-1 inhibitor to enter the U.S. market. It’s also the first FDA-approved therapy for nasopharyngeal carcinoma (NPC), a type of head and neck cancer. The drug is indicated as a monotherapy for chemo-pretreated patients or in combination with chemo for first-line treatment. In the first-line setting, Loqtorzi’s use on top of chemo cut the risk of death by 37% in the phase 3 JUPITER-02 trial. With about 2,000 new patients eligible to receive Loqtorzi each year, the drug could reach up to $200 million in peak sales in NPC alone, Coherus estimates. Coherus only holds U.S. and Canada rights to Loqtorzi, and the drug was first approved in China in late 2018. As the seventh PD-1 inhibitor to reach the U.S. market, Loqtorzi comes at a list price that’s 20% lower than Merck’s market leader, Keytruda.


Fruzaqla

Active ingredient: Fruquintinib

Disease: Metastatic colorectal cancer

Sales estimate: $340 million (2028)

Approved: Nov. 8

Company: Takeda

The scoop: As Takeda works to overcome the recent loss of exclusivity for ADHD med Vyvanse, the company is counting on new launches to deliver growth. Among the clutch of ongoing launches at the Japanese drugmaker is Fruzaqla, a colorectal cancer drug originally developed by China’s Hutchmed. Takeda picked up ex-China rights to the drug in January. In 2018, the drug became the first domestically developed cancer medicine to win approval for a major cancer type in China. In the U.S., the FDA approved the VEGF inhibitor for certain patients with metastatic colorectal cancer who’ve received two prior treatments. The FDA approval followed an August nod for Taiho and Servier’s Lonsurf in combination with bevacizumab to treat the same population. Aside from the Lonsurf combo, Takeda’s drug will go up against Bayer’s Stivarga, which generated 613 million euros in 2022. GlobalData predicts the new Takeda drug could generate $340 million by 2028. 


DefenCath

Active ingredient: Taurolidine and heparin  

Disease: Kidney disease patients on dialysis

Peak sales estimate: N/A 

Approved: Nov. 15

Company: CorMedix 

The scoop:  DefenCath is a combo treatment that reduces the risk of catheter-related bloodstream infections (CRBIs) in patients with kidney disease who are receiving hemodialysis through a catheter. It combines CorMedix’s antimicrobial and antifungal taurolidine and the anticoagulant heparin. It is the only therapy endorsed to prevent CRBIs and it is the first approval for the 17-year-old New Jersey company. Roughly a third of those who receive hemodialysis through a catheter will get a CRBI. DefenCath is the third treatment approved under the FDA’s Limited Population Pathway for Antibacterial and Antifungal Drugs. The initiative was established to facilitate the development of drugs to treat life-threatening infections in patients with unmet needs. CorMedix navigated many potholes to secure the nod as it underwent a reorganization, a CEO change, an exit from Europe, two FDA complete response letters and switches of manufacturers and suppliers.


Augtyro

Active ingredient: Repotrectinib

Disease: Non-small cell lung cancer

Sales estimate: $258 million (2027)

Approved: Nov. 15

Company: Bristol Myers Squibb

The scoop: Augtyro was the centerpiece of Bristol Myers Squibb’s $4.1 billion acquisition of Turning Point Therapeutics in 2022. The FDA’s approval allows the drug to treat ROS1-positive non-small cell lung cancer, an indication also occupied by Pfizer’s old drug Xalkori and Roche’s Rozlytrek. In this niche arena, William Blair analysts expect Augtyro could reach $258 million in sales by 2027. Back in 2022, Leerink Partners analysts said Augtyro could reach $1.4 billion in peak sales in the first line and $455 million in the second line, but they acknowledged that their estimates were more optimistic than others' projections. Augtyro’s tumor response data appeared to be the best among the three agents, and it has shown an ability to overcome a common resistance mutation called G2032R, where the rival drugs have failed.


Ryzneuta

Active ingredient: Efbemalenograstim alfa-vuxw

Disease: Neutropenia associated with chemotherapy

Peak sales estimate: N/A

Approved: Nov. 16 

Company: Evive Biotech

The scoop: Evive’s Ryzneuta treats a common side effect of chemotherapy called neutropenia, which occurs when a patient has too few of a type of white blood cells called neutrophils. By ensuring infection-fighting white blood cell counts remain high, Ryzneuta is designed to ensure patients can continue chemo treatments without interruption. Ryzneuta was originally up for an FDA nod in March. In a phase 3 study, Ryzneuta met both primary and secondary endpoints of efficacy and safety, respectively, when pitted against Amgen’s entrenched neutropenia medicine Neulasta. In the U.S., Evive has entered a commercialization pact with Aurobindo subsidiary Acrotech Biopharma to help market the drug. Aside from a split between marketing and development duties, both companies may conduct additional development of Ryzneuta to explore further opportunities, Evive and Acrotech said in a release.


Truqap

Active ingredient: Capivasertib

Disease: HR-positive, HER2-negative breast cancer

Peak sales estimate: $1.28 billion

Approved: Nov. 16

Company: AstraZeneca

The scoop: Truqap is the first AKT inhibitor to cross the FDA’s finish line, but the approval has a major limitation. The drug is allowed to be used with AZ's own Faslodex for HR-positive, HER2-negative advanced breast cancer but only in tumors that bear at least one of three gene alterations—PIK3CA, AKT1 or PTEN. The restriction came despite the drug having shown a progression-free survival benefit against Faslodex alone in a broader population, although the drug’s effect was greater in patients with those AKT-related mutations. As investigators follow the CAPItello-291 trial for its final overall survival analysis, AZ may have a chance to expand the label later, the company’s oncology business chief David Fredrickson said. Because of the limitation, Leerink analysts lowered their peak sales estimate for Truqap from $2.36 billion to $1.28 billion. AZ's Faslodex lost U.S. exclusivity in 2019, with generics to the breast cancer medicine launching that year.


Ogsiveo

Active ingredient: Nirogacestat

Disease: Desmoid tumors

Peak sales estimate: $544 million

Approved: Nov. 27

Company: SpringWorks Therapeutics

The scoop: Nearly six years after SpringWorks Therapeutics’ spinout from Pfizer, the company has its first commercial medicine in Ogsiveo. The drug treats desmoid tumors, which are noncancerous soft tissue growths that often cause severe pain and disfigurement. Historically, patients with this disease have been offered a range of treatments, including surgery, chemotherapies, radiation and tyrosine kinase inhibitors. In a phase 3 trial in mostly pretreated patients, Ogsiveo lowered the risk of disease progression or death by 71% compared with placebo. Previously known as nirogacestat, the drug is an oral gamma-secretase inhibitor that was originally developed for breast cancer. It was one of several phase 3-ready medicines that Pfizer sent off to SpringWorks as part of a 2017 spinoff and fundraising round. The drug is expected to pull in peak sales of $544 million in 2032, according to a TD Cowen analysis cited by Reuters. 


Lyfgenia

Active ingredient: Lovotibeglogene autotemcel

Disease: Sickle cell disease (SCD)

Peak sales estimate: $379 million (2029)

Approved: Dec. 8

Company: Bluebird bio

The scoop: On the same day that the FDA endorsed CRISPR and Vertex’s Casgevy to treat SCD, it also signed off on bluebird’s Lyfgenia. Both gene therapies are for patients 12 and older. While Vertex and CRISPR are charging $2.2 million for their treatment, bluebird’s gene therapy goes for $3.1 million. Despite the higher price tag, bluebird was quick to sign a large reimbursement deal with an insurer that covers roughly 100 million of the 335 million people in the U.S. Bluebird offers a three-year guarantee of Lyfgenia, providing rebates if patients are hospitalized with debilitating pain episodes (vaso-occlusion), which typify SCD. Lyfgenia’s approval was based on a trial in which 30 of 33 patients had complete resolution of vaso-occlusive events between six and 18 months after treatment. Another difference between the two SCD gene therapies is that bluebird’s has a black-box warning detailing the risk of hematologic malignancy.


Casgevy

Active ingredient: Exagamglogene autotemcel

Disease: Sickle cell disease (SCD)

Peak sales estimate: $3.9 billion

Approved: Dec. 8

Companies: Vertex Pharmaceuticals and CRISPR Therapeutics

The scoop: The FDA endorsed the long-awaited potential cure for SCD the same day that it also signed off on bluebird bio’s Lyfgenia to also treat SCD. The gene therapies are for patients 12 and older, addressing a disorder that leaves many patients dependent on a lifetime of transfusions. While Vertex and CRISPR are charging $2.2 million for the single-dose treatment, bluebird has priced Lyfgenia at $3.1 million. There is more excitement for Casgevy as it is the first medicine using the revolutionary CRISPR gene-editing system, which earned its inventors a Nobel Prize, and holds tantalizing potential to cure diseases for which there are no other treatments. Casgevy was approved based on a trial that relieved 15 of 16 (94%) patients of their debilitating pain episodes (vaso-occlusive crises) for at least 12 months. Casgevy is also up for a potential approval to treat transfusion-dependent beta thalassemia (TDT), with the FDA decision due in March of 2024.


Fabhalta

Active ingredient: Iptacopan

Disease: Paroxysmal nocturnal hemoglobinuria (PNH)

Peak sales estimate: $3.6 billion

Approved: Dec. 12

Companies: Novartis

The scoop: Dubbed a “pipeline in a pill” by ODDO BHF, the factor B inhibitor gained what Novartis hopes is the first of several approvals. Novartis is lining it up to address other complement-mediated renal and hematological diseases. Fabhalta is the first oral monotherapy approved by the FDA for PNH, a rare blood disorder. The nod covers adults who have been previously treated. C5 inhibitors, such as AstraZeneca’s Soliris and Ultomiris. Because Fabhalta acts upstream of the C5 terminal pathway, it may have an advantage in preventing blood cell destruction. Another edge is Fabhalta’s oral route of administration, versus an injection or an infusion for the C5s. The treatment will carry a boxed warning for increased risk of “life-threatening infections caused by encapsulated bacteria.” It will be made available through a Risk Evaluation and Mitigation Strategy (REMS) program that requires vaccinations for encapsulated bacteria.


Filsuvez

Active ingredient: Birch triterpenes

Disease: Epidermolysis bullosa (EB)

Peak sales estimate: N/A

Approved: Dec. 19

Companies: Chiesi 

The scoop: This was the second approval in the indication last year after Krystal Biotech scored first with topical gene therapy Vyjuvek. While Vyjuvek treats one of the two main types of the rare skin condition, dystrophic epidermolysis bullosa (DEB), Filsuvez is approved to treat DEB and junctional epidermolysis bullosa (JEB). In February of 2022, the FDA rejected Filsuvez, requesting more efficacy data. Four months later, Europe signed off on the therapy, which then was owned by Ireland drugmaker Amryt. In January of this year, Chiesi paid $1.25 billion up front to acquire Amryt. The topical gel treats the wounds associated with EB, a genetic, connective tissue disease that causes the skin to become fragile. This is the third FDA nod in 2023 for Chiesi Global Rare Diseases. The other two were for enzyme replacement therapies Elfabrio, which treats the neurological disorder Fabry disease, and Lamzede for the genetic condition alpha-mannosidosis. 


Wainua

Active ingredient: Eplontersen

Disease: Polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN)

Peak sales estimate: $700 million

Approved: Dec. 21

Companies: Ionis and AstraZeneca

The scoop: Wainua is the first auto-injector approved to treat polyneuropathy in adult patients with hereditary transthyretin-mediated amyloidosis (ATTRv-PN), a rare and debilitating disease that leads to peripheral nerve damage and motor disability within five years of diagnoses. If untreated, the disease is typically fatal within a decade. With Ionis and AZ’s option, patients can dose themselves monthly instead of traveling to specialized treatment centers. The companies look to expand to the wider ATTR cardiomyopathy patient pool, which encompasses 400,000 to 500,000 patients as opposed to ATTRv-PN’s 40,000 patients globally. Analysts at William Blair project $700 million in peak Wainua sales in just the ATTRv-PN indication. Also in the space is Alnylam’s Onpattro and newer Amvuttra, but the analysts see the market as large enough to support multiple options.


Ref: https://www.fiercepharma.com/special-reports/2023-drug-approvals-after-down-year-fda-signs-bounty-new-medicines



NextGen Class of 2024: Top Life Sciences Startups to Watch This Year

BioSpace is proud to present its NextGen Bio Class of 2024, a list of the hottest new life sciences companies in the United States. 


To come up with this list, BioSpace identified companies that launched between September 2022 and September 2023 with Series A funding. They were then assessed using several different criteria including finance, partnerships, pipeline, growth potential and innovation. After awarding points for each category, the BioSpace editorial team ranked its top 30. 

The impressive group of startups that make up BioSpace’s NextGen Class of 2024 is already making an impact on the industry. We will continue to keep a close eye on these companies as we move into the new year.


1. Rapport Therapeutics

Launched: March 2023

Location: Boston, MA, and San Diego, CA

Series A: $100M

Series B: $150M

Notable: Good rapport won this startup top billing on our list. The product of a partnership between Third Rock Ventures and Janssen Neuroscience, Rapport’s first program focuses on targeting drug-resistant seizure disorders and is in Phase I development.

Differentiator: Specificity is what sets Rapport apart, according to CEO Abe Ceesay, who spoke with BioSpace in March 2023. Current therapies for neurological disorders are nonspecific in nature, Ceesay said, while Rapport’s technology, which uses receptor-associated proteins (RAPs), has the potential to deliver highly specific therapies that are both more effective and better tolerated. The approach allows “unprecedented precision” in targeting receptors in the specific neuroanatomical regions underlying neurological disorders, according to the Series A announcement.

One More Thing: Not six months after announcing a $100 million Series A, Rapport came back with $150 million in Series B financing.


2. Apogee Therapeutics

Launched: December 2022

Location: Waltham, MA

Series A: $20M

Series B: $149M

Notable: In August 2023, Apogee dosed the first participants in a Phase I trial of lead candidate APG777, which is being developed to treat atopic dermatitis and other inflammatory diseases. APG777 is also in preclinical development for asthma.

Differentiator: Apogee is building antibodies from scratch, CEO Michael Henderson told BioSpace at the time of the company's Series B announcement in December 2022. “We are going to build brand new, novel antibodies that will provide a meaningful step change to what these patients currently have,” he said.

One More Thing: Apogee, the first spinout from Paragon Therapeutics, was one of just a few biotechs to file for an initial public offering (IPO) in 2023.


3. CARGO Therapeutics

Launched: March 2023

Location: San Mateo, CA

Series A: $200M

Notable: CARGO is another member of the select 2023 IPO club. The quickly moving startup announced its own filing in November 2023, seeking more than $315 million. The company plans to use around $220 million of the proceeds from the IPO to fund Phase II trials of its lead candidate CRG-022, an autologous CD22 CAR T cell therapy designed for patients whose large B-cell lymphoma (LBCL) is relapsed or refractory to CD19 CAR T cell therapies.

Differentiator: CARGO already has data in hand from a Phase I trial showing that treatment with CRG-022 led to durable complete responses in greater than 50% of LBCL patients in this population.

One More Thing: CRG-022 has been granted Breakthrough Therapy Designation by the FDA.


4. Aera Therapeutics 

Launched: February 2023

Location: Cambridge, MA

Series A & B: $193M

Notable: Aera launched into the sizzling hot field of gene therapy delivery with foundational technology from the lab of world-renowned scientist Feng Zhang of the Broad Institute of MIT and Harvard and the University of Utah’s Jason Shepherd.

Differentiator: Pedigree. Aera was founded by Zhang and ARCH Venture Partners. Zhang is joined by former Alnylam Oncology Head Akin Akinc, who serves as CEO, and John Maraganore, the founding CEO of Alnylam Pharmaceuticals, who serves as Aera’s board chair.

One More Thing: Aera will “explore a variety of genetic medicine modalities” and “prioritize applications based on patient impact,” Akinc told BioSpace in an email at the time of the


5. Nido Biosciences

Launched: May 2023

Location: Watertown, MA

Series A, B & Seed: $109M

Notable: Nido was founded through the 4:59 Initiative at 5AM Ventures. The company’s first clinical-stage candidate, NIDO-361, is in Phase I trials for Spinal and Bulbar Muscular Atrophy (SBMA)—also known as Kennedy’s disease—a rare, inherited neuromuscular disorder affecting men that results in a loss of skeletal muscle and motor neuron function. The disorder is caused by a genetic mutation of the androgen receptor.

Differentiator: In addition to NIDO-361, Nido is leveraging a functional genomics discovery platform based on human cell lines that uses tailored screens to identify novel therapeutic targets for myriad neuromuscular and neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and frontotemporal disorders (FTD).

One More Thing: Eli Lilly, which has a deep emphasis on neuroscience, participated in Nido’s Series A funding round.


6. ReNAgade Therapeutics

Launched: May 2023

Location: Cambridge, MA

Series A: $300M

Notable: The past three years have made clear the potential of RNA, but the aptly named ReNAgade Therapeutics wants to push the envelope and deliver RNA medicines to previously inaccessible tissues and cells.

Differentiator: Experience. ReNAgade CEO Amit Munshi was most recently the head of Arena Pharmaceuticals, which under his stewardship grew from a $300 million company into a late clinical-stage firm with nearly 40 programs before being acquired by Pfizer for $6.7 billion. Munshi is joined by CSO Peter Smith, whose resume includes leadership roles at Alnylam and Moderna.

One More Thing: Not only did ReNAgade launch with a cool $300 million, but it had also already established a joint venture with circular RNA startup Orna Therapeutics—BioSpace Class of ’22—to combine their respective platforms. A subsequent Orna-Merck collaboration is leveraging tech developed under this joint venture.


7. Orbital Therapeutics

Launched: April 2023

Location: Cambridge, MA

Series A: $270M

Notable: Orbiting in the red-hot RNA space, this startup is a spinout of Beam Therapeutics and counts John Maraganore, the founding CEO of Alnylam Pharmaceuticals, as a co-founder. Maraganore is chairman of Orbital’s board of directors.

Differentiator: Orbital is building what it calls a first-in-kind platform that combines existing and novel RNA technologies and delivery mechanisms. And it would seem the company has the cash and experience to do it.

One More Thing: Co-founder and CEO Giuseppe Ciaramella is a busy man, also serving as president of Beam. Ciaramella was previously CSO of the infectious diseases division at Moderna.


8. Abdera Therapeutics

Launched: April 2023

Location: Menlo Park, CA, and Vancouver, BC

Series A & B: $142M

Notable: The radiopharmaceuticals space quietly picked up speed in 2023, with Eli Lilly’s $1.4 billion acquisition of Point Biopharma, RayzeBio’s $311 million IPO and Abdera Therapeutics’ $142 million in combined Series A and B funds. 

Differentiator: Balance. Abdera’s website notes that radiopharmaceuticals using small ligands often fail to reach therapeutic levels inside tumors and can cause toxicities, while antibodies can limit tumor penetration and lead to systemic exposure. Abdera’s proprietary ROVEr platform allows it to strike an optimal balance between the two approaches, the company claims, with antibody-based medicines that bear a high-affinity antigen-binding domain to seek out and specifically target cancer cells.

One More Thing: Abdera’s lead asset, ABD-14, is a delta-like ligand 3-targeting molecule going after small cell lung cancer.


9. SonoThera

Launched: December 2022

Location: South San Francisco, CA

Series A: $60.75M

Notable: Eli Lilly and J&J are among the investors that believe in the potential of SonoThera’s ultrasound-guided nonviral gene delivery platform, as the two companies kicked in funds for the startup’s Series A.

Differentiator: Microbubbles. Whereas traditional approaches use viral vectors to deliver a therapeutic gene, SonoThera houses its payload in a microbubble, which is then injected into the bloodstream. The microbubble is guided to the target site using an FDA-cleared ultrasound device and a proprietary acoustic profile. This approach could allow the company to avoid the immunogenicity and high costs associated with viral vectors.

One More Thing: As of February, SonoThera had two new microbubble products to use in the development of its platform after inking an exclusive license for GE HealthCare’s Optison and Sonazoid.


10. Bitterroot Bio

Launched: June 2023

Location: Palo Alto, CA

Series A: $145M

Notable: The cardiovascular space experienced a resurgence in 2023, including the $145 million in Series A funds dropped into the coffers of Bitterroot Bio, a startup focused on the subspecialty of cardio-immunology.

Differentiator: Bitterroot’s first program targets the CD47/SIRPα pathway. While the anti-CD47 approach to cardiovascular pathology has yet to be tested in humans, blocking CD47 restored the function of macrophages in mice and facilitated the clearance of plaques and reversal of atherosclerosis, according to a 2016 study published in Nature.

One More Thing: Co-founder Irving Weissman is credited with the discovery of how tumors use the CD47 protein to evade the immune system.


11. Belharra Therapeutics

Launched: January 2023

Location: San Mateo and San Diego, CA

Series A: $130M

Notable: Backed by Versant Ventures and Roche’s Genentech, Belharra Therapeutics launched with a nine-figure funding round and a drive to advance its chemoproteomics platform.

Differentiator: Belharra’s chemoproteomics platform does not require specific amino acid residues on a protein to find and label a proteome. When the company announced its launch, it said its platform could counter the challenges in chemoprotemoics.

One More Thing: So far, the company plans to set its discovery sights in the oncology and immunology spaces, but it has also netted partnerships with Genentech and Scripps Research.


12. Kate Therapeutics

Launched: June 2023

Location: San Diego, CA

Series A: $51M

Notable: Kate Therapeutics is a biotech looking to develop AAV-based treatments for complex heart and muscle conditions.

Differentiator: Kate Therapeutics separates itself from the pack with its DELIVER platform, which uses directed evolution, RNA-based selection of capsid variants and machine learning to learn and analyze in vivo models. Kate has produced a class of capsids called MyoAAV, developed by Sharif Tabebrodbar, Kate’s CSO and co-founder.

One More Thing: Kate hit the ground running in partnerships with bigger pharma companies. Astellas inked an exclusive license to develop, produce and commercialize one of Kate’s candidates, KT430, designed to treat X-linked myotubular myopathy.


13. Convergent Therapeutics

Launched: May 2023

Location: Cambridge, MA

Series A: $90M 

Notable: The startup is seeking to develop radiopharmaceutical-based therapies to treat cancers, including prostate cancer. 

Differentiator: Convergent claims that its lead candidate in targeting prostate cancer, CONV01-α, is different from other radioactive medicines. Using the radionuclide 225Ac, the alpha particles can deliver radiation over short distances and reduce exposure to healthier tissues and cells nearby, according to the company. In a Phase I trial of the candidate, 44% of patients with metastatic castration-resistant prostate cancer experienced a decline of prostate-specific antigen levels by more than 50% after a single dose. 

One More Thing: The company attracted big-name funders such as RA Capital Management and OrbiMed. 


14. Cajal Neuroscience

Launched: November 2022

Location: Seattle, WA

Series A: $96M

Notable: Backed by Bristol Myers Squibb, Lux Capital and The Column Group, Cajal Neurosciences plans to combine human genetics and multi-omics, whole brain imaging and functional genomics to find potential drug targets.

Differentiator: A spokesperson at Cajal told BioSpace in late 2022 when the company launched that the discovery outfit uses computational genetic techniques to “identify the precise mechanism by which genetic variation mediates disease.” The company plans to build a discovery platform that allows researchers to see what is happening in a degenerating brain, leading to potential target points. 

One More Thing: Cajal is named after and inspired by Spanish neuroscientist Santiago Ramon y Cajal, who helped map cellular content and connectivity in the brain.


15. Matchpoint Therapeutics

Launched: October 2022

Location: Cambridge, MA

Series A: $100M

Notable: Matchpoint Therapeutics came on the scene with backing from Sanofi and Vertex and is focused on developing precision small-molecule medicines.

Differentiator: Its Advanced Covalent Exploration platform uses several tools such as chemoproteomics to identify new covalent binders to disease-causing proteins as well as machine learning to find targets and build out its library of covalent compounds.

One More Thing: Matchpoint’s financing is enough to see the company through 2025.


16. Bonum Therapeutics

Launched: November 2022

Location: Seattle, WA

Series A: $93M

Notable: After Roche acquired Good Therapeutics, the latter’s leadership team formed Bonum Therapeutics, which is developing a new class of “context-dependent therapeutics” that are only active when bound to a target molecule.

Differentiator: Bonum focuses on the regulation of cytokines such as IL-12, IFN-alpha and TGF-beta, with an immune-oncology focus, but the company’s technology can also have applications in other sectors. The therapeutics themselves have antibody-binding domains that can act as sensors. When bound to a target, the molecules become active.  

One More Thing: Founder John Mulligan told BioSpace in 2022 that when the deal with Roche closed, the assets that were not transferred over were folded into the new company. The company is “the same as Good Therapeutics minus one program.”


17. Juvena Therapeutics

Launched: November 2022

Location: Redwood City, CA

Series A: $41M

Notable: Launched with backing from Mubadala Capital and Horizons Ventures, Juvena Therapeutics aims to target chronic and age-related diseases.

Differentiator: Juvena uses artificial intelligence to look for proteins secreted by regenerative stem cells. It plans to use the identified proteins as drug agents and develop biologics that can target their pathways as well.

One More Thing: Juvena is still in the very early stages but already has several candidates in its pipeline for diseases such as myotonic dystrophy, sarcopenic obesity and obesity.


18. Georgiamune

Launched: August 2023

Location: Gaithersburg, MD

Series A: $75M

Notable: Backed by several investment firms, including Mubadala Capital, Alexandra Venture Investments and Catalio Capital, among others, Georgiamune already has already picked up FDA clearance for an IND for one of its candidates, a dual-function monoclonal antibody called GIM-122 to fight cancer.

Differentiator: Georgiamune’s approach centers on reprogramming immune signaling pathways to find ways to redirect the immune system to counter disease. Its strategy is to activate or down-modulate the immune system to fight cancer or autoimmune diseases.

One More Thing: The company has four additional cancer candidates besides GIM-122 and two in unspecified autoimmune diseases.


19. Nested Therapeutics 

Launched: October 2022

Location: Cambridge, MA

Series A: $90M

Notable: Nested is focused on discovering and developing precision therapies to treat cancer and has received backing from prominent financial names such as Goldman Sachs Asset Management and Cowan Healthcare Investments.

Differentiator: Nested’s platform can map mutational clusters onto the structural proteome, find “druggable pockets,” and design new drugs for those pockets, according to the company.  

One More Thing: Nested is already producing assets, with its lead compound known as NEST-1, a non-degrading dual molecular glue targeting the MAPK pathway.


20. Crossbow Therapeutics

Launched: July 2023

Location: Cambridge, MA

Series A: $80M

Notable: Eli Lilly and Pfizer Ventures have served as some of the investors in Crossbow’s Series A round. The company claims that its cancer therapies have the power and precision of a crossbow.

Differentiator: Crossbow says that its development process starts with finding a target and then uses TCR-mimetic antibodies to make an off-the-shelf T-cell engager and other immunotherapies. According to the company, its T-Bolt molecules can be adopted across a broad range of cancers.

One More Thing: Apart from big names attached to the raise, the company was founded, seeded and incubated at MPM BioImpact.


21. Ascidian Therapeutics

Launched: October 2022

Located: Boston, MA

Series A: $50M

Notable: When explaining the problem Ascidian aims to fix, then CEO Romesh Subramanian described an editor’s worst nightmare. Imagine opening a book that has been published and finding spelling and grammatical errors throughout, Subramanian said in an interview with BioSpace at the time of the Series A financing. Instead of correcting each mistake manually, he said, it would be more efficient to precisely fix whole chapters of the book. “That’s what we can do with disease. What we essentially do is use RNA to splice in and replace mutated exons with wild-type exons. If there are multiple mutations, we can drop in a large set of exons and correct it.”

Differentiator: Unlike gene therapies that target DNA, Ascidian’s approach makes these exon swaps in the RNA transcripts that encode proteins. Its lead program targets ABCA4 retinopathy, including Stargardt disease.

One More Thing: Ascidian takes its name from the sea squirt. To grow from larvae to adults, ascidians re-engineer their transcriptome through RNA trans-splicing and alternative splicing.


22. Delve Bio 

Launched: June 2023

Located: San Francisco, CA

Series A: $35M

Notable: Delve was spun out of the University of California, San Francisco.

Differentiator: Delve wants to bring the genomics revolution to infectious disease. The company’s test, Delve Neuro, samples all of the metagenomic content—RNA and DNA—within a patient’s sample to simultaneously detect bacteria, viruses, fungi and parasites.

One More Thing: Delve’s Series A financing was led by Perceptive Xontogeny Venture Fund II, which was joined in the round by Section 32 and GV.


23. Alterome Therapeutics

Launched: November 2022

Located: San Diego, CA

Series A (extended): $99M+

Notable: This San Diego–based startup is going after particularly evasive cancer targets in the “alterome,” which is made up of all the disease-driving alterations in genes.

Differentiator: Alterome’s computational chemistry platform, The Kraken, offers precise, atomic-level insights into molecular interactions, enabling ligand activity and binding mode predictions, according to the company’s website.

One More Thing: Scientific Advisory Board member Paul Workman is a precision cancer treatment pioneer, having developed the concept of the Pharmacological Audit Trail for biomarker-led drug discovery and development.


24. LinusBio

Launched: January 2023

Located: New York

Series A & A2: $24M

Notable: LinusBio is going after some pretty lofty targets with its exposome sequencing platform, including autism and ALS.

Differentiator: LinusBio’s platform introduces precision exposome biomarkers to identify a person’s various environmental exposures and potentially help diagnose diseases and monitor progression and serve as objective endpoints in clinical trials.

One More Thing: An early diagnostic candidate, StrandDx™-ASD, can assess the likelihood of autism at birth with 80% to 90% accuracy and help inform personalized treatment decisions, according to the Series A announcement.


25. Actio Biosciences

Launched: September 2023

Location: San Diego, CA

Series A: $55M

Notable: Genetics and precision medicine are a common refrain among the companies on this year’s list, but Actio is launching its development efforts with rare diseases, including Charcot-Marie-Tooth disease type 2C.

Differentiator: With its proprietary tech and deep genetics expertise, Actio is developing novel therapies that target shared underlying biology in both rare and common diseases.

One More Thing: “Defining a high-impact drug target for a common disease with a heterogenous population has a very low probability of success and has long been a major challenge for drug discovery,” Actio co-founder and CEO David Goldstein said in a statement at the time of the Series A announcement. By starting with rare diseases, the company hopes to both help patients in these indications and de-risk and guide therapeutic expansion to more common conditions, Goldstein said.


26. i2o Therapeutics

Launched: August 2023

Located: Cambridge, MA

Series A: $46M

Notable: i2o’s therapeutic focus couldn’t be hotter: The startup is researching a GLP-1 asset for type 2 diabetes in preclinical studies. This drug candidate, and several others, came with i2o’s acquisition of Intarcia Therapeutics.

Differentiator: i2o’s proprietary ionic liquids platform seeks to overcome two barriers to oral administration of biologics and peptides by protecting them from the harsh conditions in the gastrointestinal tract and assisting large molecules in crossing the epithelial lining.

One More Thing: i2o attracted the early attention of Sanofi Ventures, which took part in the company’s $4 million seed round. Both Sanofi and J&J are development partners.


27. Initial Therapeutics

Launched: May 2023

Located: South San Francisco, CA

Series A: $75M

Notable: Initial Therapeutics is another startup that bills itself on attempting to drug undruggable targets, in this case using selective termination of protein synthesis, or STOPS, to discover therapeutics.

Differentiator: Initial’s STOPS platform comprises a “bespoke suite” of technologies and capabilities, according to the company’s website. This allows the team to discover small molecules to intercept the translation of target proteins, Initial states in its Series A announcement, adding that its strategy “circumvents the need to accommodate the cellular activity of the fully formed protein or to structurally solve for docking.”

One More Thing: Initial was launched by the life sciences venture capital firm Apple Tree Partners.


28. Entact Bio

Launched: December 2022

Located: Watertown, MA

Series A: $81M

Notable: Entact Bio isn’t targeting disease-causing proteins for destruction; it’s trying to protect and enhance the function of beneficial proteins that help the body fight disease.

Differentiator: Entact’s Encompass platform uses deubiquitinases, or DUBs—enzymes that regulate proteins in cells. Enhancement-targeting chimeric (ENTAC) molecules pair beneficial target proteins with DUBs, which then shorten or remove chains of ubiquitin attached to the target protein, enhancing its function and restoring the cell to health.

One More Thing: CEO Victoria Richon was formerly founding president and CEO of Ribon Therapeutics, a clinical-stage biotech targeting stress support pathways in cancer and inflammation.


29. Rezo Therapeutics

Launched: November 2022

Located: San Francisco, CA

Series A: $78M

Notable: In Rezo’s Series A announcement, co-founder and President Nevan Krogan called the human body a “complicated mosaic of cell and molecular interactions” and said the field has been relying on a “two-dimensional” view to solve disease.

Differentiator: So, the company’s Sequence to Systems to Drugs (SSD) platform integrates data from proteomics, genetics, structural biology and chemical biology approaches. Together, these methods enable the rapid identification and study of disease-causing protein and genetic interactions, according to the announcement.

One More Thing: Co-founder and UCSF professor Kevan Shokat developed a successful approach to drugging a protein produced by the mutated KRAS gene, an achievement for which he was honored with two prestigious awards in 2023.


30. Aether Bio

Launched: August 2023

Located: Menlo Park, CA

Series A: $49M

Notable: One of the more unique companies on this list, Aether Bio is using generative AI and automation to find novel biocatalysts. With its molecular assembler platform, Aether intends to create enhanced products across the healthcare, home electronics and automotive industries.

Differentiator: Aether’s platform combines several buzzy modalities: high-throughput robotics, machine learning and synthetic biology, with the intention of mapping millions of enzyme-reaction combinations.

One More Thing: Aether’s Series A round was led by Jay Zaveri at Natural Capital and Trevor Zimmerman at Unless, an impact investment and VC firm “catalyzing the new industrial revolution.” 

Correction (Jan. 3): This story has been updated to correctly state that Alterome is based in San Diego not the Bay Area. BioSpace regrets the error.

Correction (Jan 3): This story has been updated to correctly name Convergent Therapeutics' lead asset as CONV01-α. BioSpace regrets the error. 

Ref: https://www.biospace.com/article/nextgen-class-of-2024-top-life-sciences-startups-to-watch-this-year/


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