End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.

BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its Biologics License Application (BLA) for Roctavian (valoctocogene roxaparvovec), an AAV gene therapy for adults with severe hemophilia A.

During its third-quarter earnings report, BioMarin Pharmaceutical noted that the FDA plans to hold an advisory committee meeting to discuss the company’s gene therapy for hemophilia A. 

Rare disease biotech BioMarin Pharmaceuticals Inc. announced Friday it was shedding around 4% of its global workforce, or around 120 jobs, in an effort to improve its operational efficiency.

The Institute for Clinical and Economic Review (ICER) concluded Tuesday that gene therapies for hemophilia A and B are worth it even at a hefty lifetime cost of $2.5 million.