As 2025 winds down, the pharmaceutical industry is bracing for a series of pivotal FDA decisions that could redefine treatment paradigms and reshape competitive dynamics across several major therapeutic areas.
Among the most closely watched approvals is Merck & Co.’s subcutaneous formulation of Keytruda, the blockbuster checkpoint inhibitor that has dominated oncology sales in recent years. With a PDUFA date set for September 23 and a potential launch as early as October 1, the new formulation promises more convenient administration for patients and providers. Crucially, it could also help Merck extend Keytruda’s market dominance ahead of anticipated generic competition in 2028.
Meanwhile, Novo Nordisk is awaiting a decision on its oral version of semaglutide, the active ingredient in its high-profile diabetes and obesity drugs, Wegovy and Ozempic. As the company faces mounting pressure from Eli Lilly and slowing revenue growth, the oral formulation could be a game-changer in the race to lead the GLP-1 market. The FDA is expected to make its decision in Q4.
Beyond reformulations, some companies are hoping to bring entirely new therapies to market. Stealth Biotherapeutics is facing a critical moment with its ultra-rare disease treatment, elamipretide, for Barth syndrome. After a surprising rejection earlier this year, the FDA accepted a revised application, with a decision due on September 26. Elamipretide, a first-in-class mitochondrial therapy, could become the first approved treatment for Barth syndrome, which affects only about 150 patients in the U.S.
Stealth is seeking accelerated approval based on improvements in extensor muscle strength, an intermediate endpoint. Success could set a precedent for regulatory pathways in ultra-rare diseases. Failure, however, could spell the end for the biotech, which has limited resources and is also testing the drug in other indications.
As the FDA prepares to weigh in, the final stretch of 2025 could bring transformative changes to patient care, market competition, and the future of several pharma players.
