Business
BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A
European Commission Approval Expected Q3 2022 1st Gene Therapy for Treatment of Hemophilia A Recommended for Approval in Europe More than 20,000 Adults with
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"European Commission Approval Expected Q3 2022\n1st Gene Therapy for Treatment of Hemophilia A Recommended for Approval in Europe \nMore than 20,000 Adults with Severe Hemophilia A in BioMarin Territories Across Europe, the Middle East and Africa \nConference Call Scheduled for Friday, June 24 at 11 a.m. ET\nSAN RAFAEL, Calif., June 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022.\n\n \n \n \n \n \n \n\n \nThe one-time infusion is planned to be marketed under the brand name ROCTAVIAN™ (valoctocogene roxaparvovec), for the treatment of severe hemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). Roctavian is the first gene therapy to be recommended for approval in Europe for hemophilia A. \nIt is estimated that more than 20,000 adults across Europe, Middle East, and Africa are affected by severe hemophilia A. BioMarin anticipates additional patient access through named patient sales based on an EMA approval in countries in the Middle East and Africa and expects additional market registrations to be facilitated by an anticipated EMA license.\n\"Today's positive CHMP opinion for Roctavian addresses the unmet medical needs in severe hemophilia A by providing a treatment option that has been shown in clinical studies can maintain effective levels of endogenously produced coagulation Factor VIII over multiple years with a single intravenous administration. Currently available treatment options require long-term, chronic use with a high degree of compliance to a prescribed schedule to be effective,\" said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. \"We are grateful to the patients, investigators and community who have been an integral part in reaching this important milestone that brings us one step closer to delivering on the promise and ingenuity of gene ...