- Announcing the EU approval of Roctavian for hemophilia A, BioMarin Pharmaceutical ( NASDAQ: BMRN ) disclosed its plans to resubmit the U.S. marketing application for the gene therapy to the FDA by the end of next month.
- In 2020, the agency rejected the one-time infusion, also known as valoctogene roxaparvovec, for the treatment of severe hemophilia A.
- Following conditional marketing authorization from the European Commission, BMRN gets permission to market the therapy for certain adults with severe hemophilia A with ten years of market exclusivity given its orphan drug designation.
- According to the company, Roctavian is the first approved gene therapy for hemophilia A, a blood disorder that affects more than 40,000 adults in Europe, the Middle East, and Africa. With the European approval, an estimated 3,200 patients with severe hemophilia A in 24 countries will be eligible to receive Roctavian.
- The final approval for the drug comes after an expert panel of the European Medicines Agency (EMA) endorsed its marketing authorization for hemophilia A in June.
For further details see:
BioMarin to resubmit hemophilia therapy for FDA approval after EU nod