New Data to be Presented for ROCTAVIAN from Phase 3 GENEr8-1 Study, Including Data on the Impact of ROCTAVIAN on Quality of Life and Musculoskeletal Health for Adults with Severe Hemophilia A

SAN RAFAEL, Calif., June 22, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced it will present new data from the ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy clinical trial program. These data will be shared this week in four oral presentations and two posters at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Montreal, Canada.

The U.S. Food and Drug Administration (FDA) has set a PDUFA Target Action Date of June 30, 2023, for the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy. The European Commission (EC) granted conditional marketing authorization under the brand name ROCTAVIAN on August 24, 2022.

"With our ROCTAVIAN clinical program, we are embarking on a new era in the treatment of people with severe hemophilia A," said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. "These data presented at ISTH will highlight the clinical impact of ROCTAVIAN for people living with severe hemophilia A more than three years post-infusion."

New data from the ongoing, global Phase 3 GENEr8-1 study – the largest to date for any hemophilia gene therapy – will highlight the impact of ROCTAVIAN on hemostatic efficacy, durability and quality of life of individuals with severe hemophilia A more than three years after infusion. Additional presentations will further explore the impact of ROCTAVIAN on the health and life experiences of people with hemophilia A after two years in the GENEr8-1 study using a tool called PROBE (Patient Reported Outcomes, Burdens and Experiences) and assess the impact of ROCTAVIAN on musculoskeletal health in people with severe hemophilia A.

Key ROCTAVIAN presentations at ISTH include:

Oral Presentations:

Bleeding, FVIII activity, and Safety 3 years after Gene Transfer with Valoctocogene Roxaparvovec: Results from GENEr8-1OC 20.1Sunday, June 25, 2023, 2:45 – 3 p.m., Eastern Time 

Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 TrialOC 20.2Sunday, June 25, 2023, 3 – 3:15 p.m., Eastern Time

The Impact of Gene Therapy on the Musculoskeletal Health of Patients with Severe Hemophilia AOC 20.4Sunday, June 25, 2023, 3:30 – 3:45 p.m., Eastern Time

Stable Factor VIII Expression and Improvement in Bleeding Phenotype Following Early Childhood Treatment with Adeno-Associated Viral Gene Therapy in the Severe Hemophilia A Dog ModelOC 30.2Monday, June 26, 2023, 11 – 11:15 a.m., Eastern Time

Posters:

Understanding the Requirement for Additional Factor VIII Infusion Associated with Novel Haemophilia A Treatments: An Expert Elicitation ExercisePB0633Monday, June 26, 2023, 6:30 – 7:30 p.m., Eastern Time

Quantitative Pharmacokinetic Model to Characterize and Extrapolate Long-Term FVIII Activity Levels in Patients with Severe Hemophilia A treated with Valoctocogene Roxaparvovec PB0626Monday, June 26, 2023, 6:30 – 7:30 p.m., Eastern Time

Robust Clinical Program

BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 1/2 and Phase 3 GENEr8-1 studies, the company is also conducting a single arm, open-label study to evaluate the efficacy and safety of ROCTAVIAN at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A (Study 270-303). There is also an ongoing study with the 6e13 vg/kg dose of ROCTAVIAN in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a study with the 6e13 vg/kg dose of ROCTAVIAN in people with severe hemophilia A with active or prior Factor VIII (FVIII) inhibitors (Study 270-205).

About Hemophilia A

Hemophilia A, also called FVIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective FVIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A.

People living with hemophilia A lack sufficient functioning FVIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels