The following is an announcement from BioMarin.
BioMarin announced an update to the community regarding our ongoing gene therapyÂ clinical trial program in hemophilia A. BioMarin’s investigational gene therapy forÂ hemophilia A has not been approved for use; it is in ongoing clinical trials evaluatingÂ its safety and efficacy.
Clinical Trial Overview
BioMarin’s investigational gene therapy valoctocogene roxaparvovec is currently being studied inÂ adults with severe hemophilia A.
â€¢ The first Phase 1/2 study was initiated in 2015 and consists of 15 individuals inÂ long- term follow-up, post treatment with valoctocogene roxaparvovec.
â€¢ The Phase 3 study (named GENEr8-1) is currently being conducted in 13 countriesÂ and is fully enrolled. 134 patients have received investigational gene therapy asÂ part of this study.
â€¢ The latest published data from the Phase II study was published by the New EnglandÂ Journal of Medicine in January 2020.
BioMarin extends its sincere gratitude to all study participants who haveÂ helped make this milestone possible.
In the USA, on Dec. 23, BioMarin submitted a Biologics License ApplicationÂ (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational geneÂ therapy. Subject to completion of the filing review, FDA will evaluate the data providedÂ by BioMarin and make a determination regarding the safety and efficacy. BioMarinÂ anticipates the BLA review to commence in February 2020.
In Europe; the European Medicines Agency (EMA) has validated the Company’sÂ Marketing Authorization Application (MAA) for its investigational gene therapy.Â Validation is a step where the EMA makes sure that all essential regulatory elementsÂ required for scientific assessment are included in the application. The MAA review willÂ commence in January 2020 under accelerated assessment.
No gene therapies for hemophilia have been determined to be safe or effective orÂ approved for use.
A patient’s medical team remains the best source of informationÂ regarding any health effects of hemophilia A.
For further information on BioMarin hemophilia studies, please visitÂ www.clinicaltrials.gov or contact BioMarin Medical Information atÂ firstname.lastname@example.org.