FDA to Decide on Breakthrough Gene Therapy for Adults with Severe Hemophilia A: BioMarin Provides Update on ROCTAVIAN™ (Valoctocogene Roxaparvovec)

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) received exciting news this afternoon: the U.S. Food and Drug Administration (FDA) extended its review of the company’s Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy. This groundbreaking therapy could have a life-changing impact on adults with severe hemophilia A.

The FDA has set a new PDUFA Target Action Date of June 30, 2023 for the submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study, which constitutes a Major Amendment due to the substantial amount of additional data. This amendment was previously communicated by the company.

The longest and largest study of its kind, the Phase 3 trial for a gene therapy for hemophilia featured an impressive 134 participants. This groundbreaking research is a major milestone for the treatment of this condition.

BioMarin is proud to announce the positive three-year data for its ROCTAVIAN therapy, which has the potential to revolutionize care for people with severe hemophilia A. The company has been working closely with the FDA to bring this important treatment to patients, and they are grateful for the agency’s active engagement throughout the process. Hank Fuchs, M.D., president of Worldwide Research and Development of BioMarin, expressed his enthusiasm for the data: “We believe that ROCTAVIAN has the potential to fundamentally transform care for people with hemophilia A.”

Global Regulatory Status

The FDA recently wrapped up a thorough Pre-License Inspection of the manufacturing facility in early December 2022, providing a detailed assessment of the facility’s capability and readiness to produce quality products.

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, a groundbreaking regenerative medicine therapy designed to address an unmet medical need in patients with serious conditions. This RMAT designation complements the Breakthrough Therapy designation that the company received for valoctocogene roxaparvovec in 2017, and is part of an expedited program that facilitates the development and review of such therapies.

BioMarin’s valoctocogene roxaparvovec has been granted special designations by the FDA and EMA due to its ability to treat severe hemophilia A. It has been awarded the RMAT designation and Breakthrough Therapy designation, as well as Orphan Drug designation from both the FDA and EMA. On August 24, 2022, the European Commission also granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN. This gene therapy is providing hope to those suffering from rare, life-threatening, or chronically debilitating diseases.

Robust Clinical Program

BioMarin is pushing the boundaries of gene therapy with its comprehensive program to treat severe hemophilia A. From the global Phase 3 study GENEr8-1 to the ongoing Phase 1/2 dose escalation study, BioMarin is also conducting a Phase 3, single arm, open-label study to evaluate the safety and effectiveness of valoctocogene roxaparvovec administered at a dose of 6e13 vg/kg with prophylactic corticosteroids. Additionally, ongoing Phase 1/2 studies are underway to assess the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies and active or prior Factor VIII inhibitors. These groundbreaking studies are bringing us one step closer to finding a cure for hemophilia A.

About Hemophilia A

Hemophilia A is a serious disorder that affects 1 in 10,000 people worldwide. Also known as Factor VIII deficiency or classic hemophilia, this X-linked genetic disorder is caused by a missing or defective Factor VIII, a vital clotting protein. While it’s usually passed down from parents to children, in one-third of cases, the mutation is spontaneous – a new mutation that wasn’t inherited. With the right care and treatment, people with hemophilia A can lead full and active lives.

Living with hemophilia A can be life-altering. Those with the most severe form of the disease experience painful, spontaneous bleeds into their muscles or joints, and make up approximately 50% of the hemophilia A population. To manage the condition, individuals must undergo frequent intravenous Factor VIII infusions or bispecific monoclonal antibody injections to mimic the activity of Factor VIII. However, many still experience breakthrough bleeds, leading to progressive and debilitating joint damage, and drastically reducing their quality of life.

About BioMarin

BioMarin is a pioneering biotechnology company that has been empowering lives since its foundation in 1997. Through revolutionary genetic research and development, the company has successfully developed and commercialized targeted therapies that directly address the root causes of genetic conditions. With eight revolutionary therapies currently on the market, BioMarin has demonstrated its commitment to providing innovative solutions for rare genetic disorders. Their cutting-edge drug discovery process has created a vast and diverse pipeline of pre-clinical, clinical, and commercial candidates, enabling them to be the first to market or provide a unique benefit compared to existing treatments.

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