Regeneron’s Cemdisiran Accepted for FDA Priority Review in Myasthenia Gravis

Novo Nordisk said the European Medicines Agency's Committee for Medicinal Products for Human Use has issued a positive opinion recommending approval of denecimig, also known as Mim8, for the treatment of hemophilia A, with or without inhibitors, in adults and children. Denecimig is a next-generation factor VIIIa mimetic bispecific antibody given subcutaneously as routine prophylaxis, with flexible dosing that includes once-monthly, every-two-week and weekly administration, and it is being developed as a prefilled pen. Novo expects to launch the drug in the first European countries in the fourth quarter of 2026 under the brand name Frehemgo, followed by a broader rollout across the European Union beginning in early 2027. The positive opinion is supported by data from the FRONTIER clinical program, which comprises five studies evaluating denecimig as a prophylactic treatment for hemophilia A across pediatric and adult populations, with or without inhibitors; in the FRONTIER 2 study denecimig significantly reduced annualized bleeding rates, results in children younger than 12 in FRONTIER 3 were consistent with those in adolescents and adults, and in FRONTIER 5 no new safety concerns were identified when participants switched directly from emicizumab to denecimig. The candidate is also under regulatory review in the United States for routine prophylaxis in adults and children with hemophilia A, with or without inhibitors, after Novo submitted a biologics license application to the FDA in September 2025. Novo already markets Alhemo for hemophilia A and B in patients with or without inhibitors.