New Seven-Year Follow-Up Phase 2 Study Results Showed Durable Hemostatic Benefit for Adults with Severe Hemophilia A

SAN RAFAEL, Calif., Feb. 6, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that new data on ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) will be presented at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress, Feb. 6-9, 2024.

Results from a Phase 2, open-label study of ROCTAVIAN showed that median factor VIII (FVIII) activity at year seven remained in the mild hemophilia range (10.3 IU/dL per chromogenic assay), and mean annualized bleeding rate (ABR) for treated bleeds over the full follow-up period decreased by 96% from baseline for adults with severe hemophilia A in the 6x1013 vg/kg dose cohort. The majority of participants maintained hemostasis, with only two of the seven participants in the study returning to regular prophylaxis, and no new safety signals emerged. These data will be presented as a poster presentation from 6:30-7:30 p.m. Central European Time (CET) on Wednesday, Feb. 7.

"People living with severe hemophilia A face a lifelong treatment burden, including frequent injections or infusions and a high risk of health complications like uncontrolled bleeding and irreversible joint damage, which may persist despite good adherence to prophylactic therapy," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We are pleased to present data showing the impact of one-time treatment with ROCTAVIAN over seven years following the infusion, underscoring the potential of gene therapy to make a meaningful and long-lasting impact for people living with severe hemophilia A."

Additional studies will be shared during oral presentations on Friday, Feb. 9 from 8:30-10:00 a.m. CET, including preliminary results from GENEr8-INH, a Phase 1/2 study evaluating the safety and efficacy of ROCTAVIAN in participants with active and prior FVIII inhibitors, as well as one-year results from GENEr8-3, a Phase 3b study evaluating the use of prophylactic corticosteroids with ROCTAVIAN treatment.

BioMarin's key presentations at EAHAD include:

Poster Presentations:

Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A

#PO124

Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET

Treatment preferences in people with severe haemophilia A: a discrete choice experiment in the United States

#PO314

Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET

Health-related quality of life and long-term joint damage in people with severe haemophilia A in Brazil

#PO144

Wednesday, Feb. 7, 2024, 6:30-7:30 p.m. CET

Oral Presentations:

Safety and efficacy of valoctocogene roxaparvovec in participants with active and prior FVIII inhibitors: preliminary results from GENEr8-INH, a phase 1/2 study

#OR10

Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET

Safety and efficacy of valoctocogene roxaparvovec with prophylactic corticosteroids: 1-year GENEr8-3 results

#OR02

Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET

Human liver biopsy analysis reveals lower RNA transcription may contribute to a decline in FVIII levels following AAV5-hFVIII-SQ gene therapy

#OR06

Friday, Feb. 9, 2024, 8:30-10:00 a.m. CET

About Hemophilia A

Hemophilia A, also called factor VIII (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 one-third of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A.