BioMarin: Clinical Development Program Update

Update for Hemophilia Associations

BioMarin is pleased to update the community regarding our 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.
BioMarin Hemophilia Update For Patient Associations View PDF | Download PDF

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 involved 15 individuals and two 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. 134 patients have received investigational gene therapy as part of this study.
On May 19, 2021, BioMarin released a high-level update on the Phase 1 / 2 study of valoctocogene roxaparvovec, including the most recent results based on 5 years of follow up data for individuals receiving the 6E13 vg/kg dose.
BioMarin intends to present more detail on the five-year update from the ongoing Phase 1 / 2 study during an oral presentation at the upcoming International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress taking place July 17-21.
BioMarin extends its sincere gratitude to all study participants who have helped make this milestone possible.

Regulatory Status

In the United States, BioMarin plans to submit two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec. BioMarin is targeting a Biologics License Application (BLA) submission in the second quarter of 2022 assuming favorable study results, followed by an expected six-month review procedure by the FDA.
In Europe, BioMarin plans to submit a Marketing Authorization Application (MAA) for valoctocogene roxaparvovec for the treatment of severe hemophilia A with one-year results from the Phase 3 GENEr8-1 study to the European Medicines Agency (EMA) in June 2021.

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 medinfo@bmrn.compage1image42713472page1image42713664

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BioMarin Hemophilia Update For Patient Associations View PDF | Download PDF