BioMarin to Resubmit Gene Therapy Roctavian for EU Approval in June

BioMarin to Resubmit Gene Therapy Roctavian for EU Approval in June

295088

BioMarin to Resubmit Gene Therapy Roctavian for EU Approval in June

The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, BioMarin’s regulatory applications for Roctavian (valoctocogene roxaparvovec) got rejected both in Europe and in the U.S., with regulatory agencies requesting longer follow-up data from the Phase 3 GENEr8-1 trial (NCT03370913), which is investigating the efficacy of the one-time therapy in 134 patients. Accelerated assessment is…

You must be logged in to read/download the full post.