WASHINGTON (dpa-AFX) - BioMarin Pharmaceutical Inc. (BMRN) announced the FDA approved ROCTAVIAN gene therapy for the treatment of adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. The company noted that the one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S.
The company said the FDA approval is based on data from the global Phase 3 GENEr8-1 study, the largest Phase 3 trial of any gene therapy in hemophilia. BioMarin will conduct an extension study that will follow all clinical trial participants for up to 15 years, and post-approval studies to follow those dosed in a real-world setting for 15 years or more.
The European Commission granted conditional marketing authorization to ROCTAVIAN on August 24, 2022.
For More Such Health News, visit rttnews.com.
Copyright(c) 2023 RTTNews.com. All Rights Reserved
Copyright RTT News/dpa-AFX