BioMarin Announces Gene Therapy Program Updates

Industry News
The following is an announcement from BioMarin.

BioMarin announced an update to the community regarding our ongoing gene therapy clinical trial program in hemophilia A. 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.
Clinical Trial Overview
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 (named GENEr8-1) is currently being conducted in 13 countries and is fully enrolled. 134 patients have received investigational gene therapy as part of this study.
• The latest published data from the Phase II study was published by the New England Journal of Medicine in January 2020.
BioMarin extends its sincere gratitude to all study participants who have helped make this milestone possible.
Regulatory Status
In the USA, on Dec. 23, BioMarin submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational gene therapy. Subject to completion of the filing review, FDA will evaluate the data provided by BioMarin and make a determination regarding the safety and efficacy. BioMarin anticipates the BLA review to commence in February 2020.
In Europe; the European Medicines Agency (EMA) has validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy. Validation is a step where the EMA makes sure that all essential regulatory elements required for scientific assessment are included in the application. The MAA review will commence in January 2020 under accelerated assessment.
No gene therapies for hemophilia have been determined to be safe or effective or approved for use.
A patient’s medical team remains the best source of information regarding any health effects of hemophilia A.
For further information on BioMarin hemophilia studies, please visit or contact BioMarin Medical Information at