BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community

BioMarin is committed to updating the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A 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 fully enrolled and currently being conducted in 13 countries
with 134 patients that have received investigational gene therapy.

Regulatory Status:
On December 23, 2019, BioMarin submitted a Biologics License Application (BLA) to the U.S. Food and
Drug Administration (FDA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults
with hemophilia A.

On August 18th, 2020, BioMarin received a Complete Response Letter from the FDA. The FDA issues
Complete Response Letters to indicate that the review cycle for an application is complete and that the
application is not ready for approval in its present form. The FDA did not identify any new safety
concerns but recommended that BioMarin complete the Phase 3 Study and submit two-year follow-up
safety and efficacy data on all study participants.

BioMarin remains committed to collaborating with the FDA to address points raised during the BLA
review and generating additional data to support the FDA’s benefit-risk evaluation of valoctocogene
roxaparvovec.

The FDA granted valoctocogene roxaparvovec Breakthrough Therapy and orphan drug designations.
The European Medicines Agency (EMA) review of the company’s marketing authorization (MAA) for
valoctocogene roxaparvovec is ongoing.

BioMarin extends sincere gratitude to our investigators and the study participants who have
contributed to the investigational study thus far.

A patient’s medical team remains the best source of information regarding any health effects of
hemophilia A.

DRAFT ©2020BioMarin pharmaceutical, Inc. | biomarin.com

For additional information on BioMarin clinical studies:

  • Visit www.clinicaltrials.gov and type in the study code BMN 270
  • For inquiries or to provide feedback from advocacy organizations, please contact patientadvocacy@bmrn.com
  • Contact BioMarin Medical Information toll free at 800.983.4587 or medinfo@bmrn.com