In 2024, the FDA approved 50 novel drugs, a slight decrease from the 55 approved in the previous year. First-in-class therapies accounted for a significant portion of approvals, including donidalorsen, developed by Ionis Pharmaceuticals for hereditary angioedema (HAE), and rezdiffra, from Karuna Therapeutics, for metabolic-associated steatohepatitis (MASH).
The US Food and Drug Administration announced major changes to ease regulatory roadblocks for low-cost copycat versions of high-priced biologic drugs. Known as biosimilars, these drugs are considered crucial to driving down drug prices and have been slow to enter the market due to patent challenges from brand-name makers. The FDA says its new rules would help speed up the development process.
Unlike traditional chemically-derived medicines, biologics are made from living cells and have more complex manufacturing processes, making them a more expensive development route. Although the FDA has already approved 76 biosimilars, they make up only a small fraction of all approved biologic medications. The agency says that, on average, they cost half as much as the branded product they are similar to.
While some approvals have gone forward normally since April, when a 20% reduction in workforce at the FDA reduced its drug review staff by nearly 3,000, signs are emerging that the Trump administration’s approach to drug regulation may be more restrictive than in the past. That could mean fewer drug approvals and delayed decisions on clinical trials, experts warn. The FDA’s decision to delay a hearing for a rare disease drug from Stealth BioTherapeutics, for example, follows an earlier denial of a request from Vanda Pharmaceuticals for a similar treatment.