Fierce Pharma - Small Company FDA Drug Approvals 20242024 drug approvals: Small companies loom large with several key FDA nods By Kevin Dunleavy - Jan 2, 2025 12:00am Comparing the FDA new drug approval lists from 2023 and 2024 reveals a significant difference in the size of the companies that achieved the regulatory triumphs.
In 2023, among the 68 FDA approvals highlighted in Fierce Pharma’s annual report, 43 (63%) were sponsored or co-sponsored by companies that generated at least $3 billion in revenue in 2023. That stands in contrast to the 2024 approval list, which shows that among 55 FDA nods, just 23 (42%) were sponsored or co-sponsored by companies with sales that reached $3 billion in 2023.
Why the sudden shift?
The change reflects a trend in which biotechs have become more resistant to buyout offers from larger companies. Smaller companies also are showing more willingness to market their products on their own as opposed to bringing on a Big Pharma partner.
In 2024, only two approvals were gained by companies that had lined up a commercial partner. Those were achieved by Syndax with Incyte and Genfit with Ipsen. In 2023, there were 11 such collaborations on the date of their approval.
It was a year highlighted by the ability of small companies to score with the FDA. Consider the less-than-household names of the first eight companies that gained new drug approvals in 2024: Ligand, Iovance, Allecra, Hugel, BeiGene, Madrigal, Kyowa Kirin and Idorsia. Similarly, near the end of the year there was run of approvals from the likes of Iterum, Autolus, PTC, Syndax, Jazz, BridgeBio, Merus, Checkpoint, Neurocrine, Betta, Mesoblast and Ionis.
In 2024, no company gained more than two approvals. It was a major shift from 2023 when Pfizer scored a remarkable seven nods and three other major companies—GSK, Biogen and UCB—secured three each.
Small companies come up big
In addition to the sheer number of approvals, biotechs accounted for several of the most significant FDA endorsements of 2024. After a decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis, for example, Madrigal scored with Rezdiffra, which Evaluate projects to achieve sales of $2.1 billion in 2030.
It’s been a long wait as well for a new treatment for chronic obstructive pulmonary disorder. Verona answered the call with Ohtuvayre, which Evaluate pegs to generate $1.5 billion in revenue in 2030. Ascendis Pharma gained a nod for Yorvipath, which becomes the first product in the U.S. for hypoparathyroidism in adults and is expected by Evaluate to achieve sales of $1.8 billion in 2030.
Evaluate also projects sales of $1.7 billion in 2030 for a pair of new drugs from small companies—Galderma’s prurigo nodularis treatment Nemluvio and ImmunityBio’s cancer immunotherapy Anktiva.
Other products brought by small companies that could achieve blockbuster sales by 2030—according to Evaluate—include Geron’s blood cancer treatment Rytelo and Day One’s brain tumor therapy Ojemda.
Little Syndax runs with big dogs
Most of the drugmakers that scored multiple approvals in 2024 were the usual Big Pharma suspects—Eli Lilly, Merck, Pfizer and Roche. But there was an under-the-radar company that also garnered a pair of nods.
Syndax, a 19-year-old Massachusetts biotech which had just 107 employees at the start of 2023, gained its two approvals three months apart. In August, the FDA gave Syndax and its commercial partner Incyte a thumbs-up for Niktimvo to treat chronic graft-versus-host disease. Then, in November, Syndax scored with Revuforj, the first menin inhibitor to treat a genetic type of leukemia called lysine methyltransferase 2A.
As for the other companies with multiple approvals, Merck cashed in with a pair of important nods. Winrevair, which is the first disease-modifying treatment for pulmonary arterial hypertension, has been projected by Evaluate to generate $3 billion in revenue in 2030. Merck’s other nod came for Capvaxeve, the first pneumococcal vaccine designed specifically for adults.
Roche’s approvals were for drugs that the company projects can each generate peak sales of 2 billion Swiss francs ($2.3 billion). Itovebi is a treatment for HR-positive, HER2-negative breast cancer, while Piasky was endorsed to treat paroxysmal nocturnal hemoglobinuria.
Pfizer’s FDA nods came for a pair of hemophilia treatments. Hympavzi, an anti-tissue factor pathway inhibitor, was approved for hemophilia A and B, while gene therapy Beqvez is for hemophilia B.
Key Big Pharma approvals
Of the new drug approvals that the FDA handed out in 2024, two of the most important came in the third quarter and both were for neurological conditions. In July, the agency signed off on Eli Lilly’s Alzheimer’s disease treatment Kisunla. Then, in September, the FDA endorsed Bristol Myers Squibb’s schizophrenia drug Cobenfy.
Both are for disorders where patients had been waiting for new treatment options. And both are for conditions where millions suffer but only a small percentage are treated.
Part of the excitement for Kisunla is that it's the first anti-amyloid drug that allows for stopping therapy when amyloid plaques are removed, enabling fewer infusions and lower treatment costs.
Meanwhile, the buzz for Cobenfy comes as it is the first medicine for schizophrenia with a new mechanism of action in seven decades. Since 1954, when chlorpromazine reached the market, there have been improvements on the treatment, but all have been from the dopamine receptor class.
Oddly enough, both Kisunla and Cobenfy originated in the labs of Lilly. While Kisunla is a homegrown product, Cobenfy traces its roots to Lilly’s discovery of xanomeline more than three decades ago. After Lilly gave up on the compound because of its harsh side effects, Karuna gained its rights in 2012 and turned it into gold. In December 2023, BMS bought out Karuna for $14 billion.
While 2024 was a stellar year for Lilly as its market cap continued to spiral upward, the company also excelled on the approval front as it also won a nod for eczema treatment Ebglyss, which is tabbed by GlobalData to achieve sales of $2.8 billion in 2030.
Pricey cell and gene therapies
Entering 2024, the most expensive drug in the U.S. was CSL Behring and uniQure’s hemophilia gene therapy Hemgenix at $3.5 million. But that figure was eclipsed last year—and not by a small margin.
The new most expensive drug is Kyowa Kirin’s Lenmeldy, which goes for $4.25 million, for children with metachromatic leukodystrophy. In November, PTC Therapeutics scored approval for Kebilidi, a treatment for the ultrarare disease aromatic L-amino acid decarboxylase deficiency, which has yet to be priced in the U.S. In the U.K., where it was approved in 2022 under the commercial name of Upstaza, a dose goes for $3.7 million.
Pfizer’s gene therapy Beqvez is priced at $3.5 million, matching the figure for Hemgenix, as both treat the same patient population.
The pricing escalation also applied to new cell therapies as Adaptimmune is charging $727,000 for Tecelra, making it the costliest cell therapy ever. Tecelra is the first engineered cell therapy for a solid tumor and the first T-cell receptor treatment to enter the market.
Another landmark cell therapy approval was had by Iovance with Amtagvi, which became the first individualized tumor-infiltrating lymphocyte therapy to reach the market.
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.
See link for detailed list of 55 FDA drug approvals:
https://www.fiercepharma.com/pharma/2024-drug-approvals