BioMarin Pharmaceutical Inc. has announced a progress update on its Biologics License Application (BLA) for the valoctocogene roxaparvovec adeno-associated virus gene therapy for adults with severe hemophilia A.

The US Food and Drug Administration (FDA) scheduled its prelicensure inspection of BioMarin’s therapy manufacturing facility and requested submission of the upcoming 3-year data analysis from the phase 3 GENEr8-1 study, company officials said.

“We appreciate the level of engagement from the FDA this early in their BLA review cycle and are pleased to share that the inspection of our gene therapy manufacturing facility has now been scheduled. Additionally, with their request related to the upcoming three-year data analysis from our Phase 3 GENEr8-1 study, FDA stated that these data are expected to provide longer-term efficacy and safety information and would thus be useful to people with Hemophilia A and healthcare providers in making better and more informed decisions when considering valoctocogene roxaparvovec as a treatment choice should it be approved,” stated Hank Fuchs, MD, president of worldwide research and development at BioMarin.  

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The FDA has not yet announced any changes in the target date by which it must respond to BioMarin’s BLA, currently set as March 31, 2023, although a 3-month extension might be granted if the data submission is deemed a major amendment. 

A yet-to-be scheduled advisory committee meeting might be an opportunity for the pharmaceutical company to further discuss the 3-year data analysis.

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“We are encouraged by the bleed control results observed in the first cohort of patients who reached three years of observation in the Phase 3 study as shared in January, and we look forward to reviewing the three-year results from all participants. We anticipate sharing the three-year results from all 134 participants in our Phase 3 study in early 2023,”  Fuchs added. 

BioMarin’s gene therapy for Hemophilia A received the RMAT Designation and Breakthrough Therapy Designation, as well as the orphan drug designation from the EMA and FDA. The company’s comprehensive gene therapy program for the treatment of severe hemophilia A includes multiple clinical studies evaluating the safety and efficacy of the treatment in various modalities of patients with Hemophilia A.


BioMarin announces incremental progress on biologics license application (BLA) review for valoctocogene roxaparvovec AAV gene therapy for adults with severe hemophilia A program. News release. BioMarin Pharmaceutical Inc.; November 7, 2022.