This section contains news regarding the Food and Drug Administration’s consideration of new drugs, therapies and treatments. It includes approvals, denials, appeals and resubmissions.
FDA Commissioner Marty Makary has talked about the need to shorten the agency’s approval process and discussed artificial intelligence (AI) as a tool to help do so. He has also promised to use vouchers to speed up reviews for certain drugs, which could be a boon to companies developing products that address health crises or deliver “innovative cures.” But turbulence at the FDA is causing uncertainty about how Makary will approach the program.
2025’s first half has seen an avalanche of novel drugs approved across a wide range of specialties, from rare diseases to oncology. Ionis Pharmaceuticals’s nedosiran was the most notable approval, as its treatment for hereditary angioedema (HAE) improves on its predecessor Trikafta by having more convenient dosing and broader eligibility. Vaccines were another area of strength, with nine approvals this year (five mRNA vaccines and four oligonucleotides), including mRNA maker Moderna’s mRNA-based RNAi cancer therapy XmAb-155, which is expected to reach the market in 2022.
Other noteworthy drugs included Gilead’s twice-yearly shot for HIV prevention and Nuvation Bio’s ROS1 inhibitor for non-small cell lung cancer. Several other first-in-class drugs were approved, including Novo Nordisk’s GLP-1/GIP/GCGR triple receptor agonist retatrutide for weight loss and a glucagon antagonist/GLP-1 agonist combination for type 2 diabetes. The final big approval was from Verona Therapeutics for otuvadrisib, an oral RAF inhibitor that is the first drug to treat a rare form of non-small cell lung cancer.