While the year’s tally of green lights from the FDA’s Center for Drug Evaluation and Research (CDER) is lower than the previous year’s, 2024 saw several long-awaited therapies reach the finish line. Oncology remained the single largest focus area among last year’s endorsed therapies and as opposed to 2023 when Pfizer scored a total of seven thumbs up, in 2024 there was no clear Big Pharma winner and many approvals were granted to lesser-known players in the biotech arena.
Oncology Leads the Roster
2023 may have seen a record-breaking 55 novel drug approvals, the highest since 2018, but with its full list of authorisations reaching 50, 2024 was also an impressive year that gave the go-ahead to innovative therapies in a range of therapeutic areas. Cancer treatments took the lead with 15 approvals, including a number of first-in-class treatmensts and a new PD1/PDL1 blocker. With six approvals each, dermatology and non-malignant haematology followed while neurology and infectious diseases saw fewer approvals than in previous years.
Among last year’s novel therapies, most were small molecules with 32 approvals, followed by proteins with 16.
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Long-Awaited Breakthroughs
2024 also brought novel medicines to disease areas that have long suffered from a dearth of innovations. One of the most momentous FDA nods last year was Cobenfy from Bristol Myers Squibb, the first novel treatment for schizophrenia in 35 years. The drug, whose development stems all the way back to the 1990s, was originally developed by Karuna Therapeutics. BMS had the presence of mind to pick up Cobenfy, a fixed-dose combination of xanomeline and trospium originally known as KarXT, for USD 14 billion just when it was reaching the finish line. “This approval offers a new alternative to the antipsychotic medications people with schizophrenia have previously been prescribed,” said Tiffany Farchione, director of the Center for Drug Evaluation and Research’s psychiatry division, office of neuroscience, at the time of approval.
The FDA’s endorement of Eli Lilly’s Alzheimer’s disease therapy, Kisunla, also represented a big step for a field in urgent need of treatment options. The once-monthly treatment is the first of its kind – an amyloid plaque-targeting therapy – with evidence to support stopping therapy when amyloid plaques are removed, which means that it stands to lower treatment costs. “I am encouraged by the potential to stop treatment, which could reduce out-of-pocket costs and infusion burden for eligible patients,” Howard Fillit, co-founder and chief science officer at the Alzheimer’s Drug Discovery Foundation (ADDF) said of the drug.
Another area where innovative medicines have been long-awaited is nonalcoholic steatohepatitis (NASH), a disease that affects some 10-15 million people in the US. No treatment was available to patients until Madrigal Pharmaceuticals’s Rezdiffra became the first and only FDA-approved NASH medication in March of last year. NASH, also known as metabolic dysfunction-associated steatohepatitis (MASH), involves the accumulation of fat and inflammation in the liver, which causes scarring and may lead to liver failure. “This is a day of celebration for patients with NASH who have been waiting many years for the first approved therapy,” Wayne Eskridge, co-founder and CEO of the Fatty Liver Foundation, said of the approval.
Cell & Gene Therapy Ground-Breakers
2024 also saw some landmark approvals in the field of cell and gene therapies, namely Pfizer’s one-time gene therapy for adults with Hemophilia B, a rare genetic bleeding disorder that prevents normal blood clotting due to a deficiency in FIX. “Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions,” said Adam Cuker, Director of the Penn Comprehensive and Hemophilia Thrombosis Program. “A one-time treatment with BEQVEZ has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term.”
Another milestone autorisation was for Amtagvi from Iovance Biotherapeutics, the first FDA-approved T-cell therapy for solid tumor cancer and the first treatment option for advanced melanoma after anti-PD-1 and targeted therapy. Like other CAR-T cell therapies, Amtagvi is manufactured using patients’ immune cells, which are collected from a portion of their tumor, expanded outside the body, and infused back into the patient.
Smaller Players Reach the Finish Line
As opposed to 2023 when Pfizer scored a total of seven novel drug thumbs up, in 2024 there was no clear leader among Big Pharma and several lesser-known companies like Ligand, Iterum, or BridgeBio, achieved FDA endorsement for their innovative therapies. Whereas pharma giants Merck, Pfizer, Roche and Lilly landed no more than two approvals each, a string of biotechs reached the home stretch with one approval. Syndax, a small Massachusetts-based biotech, achieved two go-aheads with its chronic graft-versus-host disease treatment, Niktimvo, and the first menin inhibitor to treat leukemia type lysine methyltransferase 2A, Revuforj.
And, for the most part, the biotechs who made the grade this year did it on their own, without a pre-exisiting commercial partner. Just two approvals were gained by companies who had already put a commercialisation deal in place: Syndax with Incyte and Genfit with Ipsen.
Full list of approvals:
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