BioMarin Hemophilia A Clinical Development Program Update

Industry News
The following is a press release from BioMarin.

 BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. 
Clinical Trial Update 
BioMarin’s investigational gene therapy valoctocogene roxaparvovec, is currently being studied in adults with severe hemophilia A. 
The first Phase 1/2 study was initiated in 2015 and consists of 15 individuals in long- term follow-up, post treatment with valoctocogene roxaparvovec. 
The Phase 3 study is currently being conducted in 13 countries and 130 patients have been enrolled and have received investigational gene therapy. 
Regulatory Status 
The European Medicines Agency recently granted BioMarin’s accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. Additionally, the Food and Drug Administration has granted valoctocogene roxaparvovec Breakthrough Therapy designation. Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the EMA. 
BioMarin plans to submit marketing applications in United States and Europe before the end of 2019 which will allow regulators to evaluate the safety and efficacy of valoctocogene roxaparvovec. 
BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. 
A patient’s medical team remains the best source of information regarding any health effects of hemophilia. 
For further information on BioMarin hemophilia A studies, visit or contact BioMarin Medical Information at