Tag Archive for: Roctavian

Activist investor Elliott Investment Management has bought more than a $1 billion stake in BioMarin Pharmaceutical, according to an exclusive report from Reuters published Tuesday, citing two unnamed sources familiar with the matter.

BioMarin Pharmaceutical Inc. announced that an individual in Germany with severe hemophilia A was treated with Roctavian (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been given commercially in Europe.

After an initial rejection, BioMarin has finally secured the FDA’s approval for Roctavian, the first gene therapy in the U.S. for the most common form of the bleeding disorder.

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.