BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. BioMarin鈥檚 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鈥檚 valoctocogene roxaparvovec, is currently being studied in adults with severe hemophilia A. The first Phase 1/2 study was initiated in 2015 and involved 15 individuals and four dose levels. These individuals are now 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 with 134 patients having received investigational gene therapy as part of this study.

Regulatory Status

On March 8, 2021, BioMarin announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec.

RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which BioMarin received in 2017, allowing early, close, and frequent interactions with the FDA.

One additional feature of the RMAT program is that sponsors of products that have been granted RMAT designation and which receive accelerated approval may be able to fulfill the post-approval requirements from clinical evidence obtained from sources other than the traditional confirmatory clinical trials.

In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin’s valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

BioMarin extends its sincere gratitude to our investigators and the study participants who have helped contribute to our investigational gene therapy program.

For More Information:

  • Visit and type in the study code BMN 270
  • For inquiries or to provide feedback from advocacy organizations, please contact
  • Contact BioMarin Medical Information at 1-800-983-4587 or

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