BioMarin's Roctavian gets FDA action date extended to end of June
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- The US FDA has told BioMarin Pharmaceutical (NASDAQ:BMRN) that it is extending the action date on the company's Biologics License Application for Roctavian (valoctocogene roxaparvovec) to June 30.
- The FDA said that a three-year data analysis provided by the company from the ongoing pivotal GENEr8-1 study constituted a Major Amendment because of significant new data.
- The study enrolled 134 individuals with hemophilia A.
- Roctavian is a gene therapy for adults with severe hemophilia A. It is already approved in Europe.