Aug. 24 Quick Takes: EC approves BioMarin’s hemophilia A gene therapy
Plus Ovid, Gensaic in gene therapy deal and updates from Digital Diagnostics, BioInnovation Institute, Moderna, Relief and more
In a milestone for severe hemophilia A patients, the EC has granted conditional approval to Roctavian valoctocogene roxaparvovec from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to treat the disease and endorsed EMA’s recommendation that the gene therapy maintain orphan drug designation, giving it a 10-year period of market exclusivity. The company said it plans to submit a BLA to FDA for Roctavian, a one-time infusion that delivers a functional gene to enable the body to produce Factor VIII on its own without the need for continued hemophilia prophylaxis, by the end of September.
Roctavian’s approval makes two drugs approved in Europe ahead of the U.S. in a day. The EC also granted conditional marketing authorization to Tecvayli teclistamab, a first-in-class CD3 x BCMA bispecific from Johnson & Johnson (NYSE:JNJ). ...