Biomarin limits marketing of its FVIII gene therapy, Roctavian

SAN RAFAEL, California, August 5, 2024 — Biomarin has announced that it will limit commercial, research and manufacturing programs for its factor VIII gene therapy valoctocogene roxaparvovec (Roctavian) to three countries—United States, Germany and Italy—where the treatment has already received regulatory approval and a reimbursement pathway.

Moreover, no more patients will be dosed in ongoing trials and no new investigational studies will be started. Biomarin will continue to support and monitor patients who have already received valoctocogene roxaparvovec in clinical trials.

Valoctocogene roxaparvovec has not been reviewed by Health Canada and it now appears that plans to introduce this therapy in Canada are suspended indefinitely.

While 134 people with severe hemophilia A received valoctocogene roxaparvovec in the Phase 3 clinical trial, only a very small number have been treated commercially in the U.S, Germany and Italy since it was first approved by the European Medicines Agency in August 2022 and by the U.S. Food and Drug Administration in June 2023.

The principal concern with the therapy has been factor VIII expression levels dropping over time. Roctavian has shown a drop of 14-27% (FVIII IU/ml) year-on-year from peak levels, although individual responses vary greatly.

For more information on gene therapy, please see the CHS Gene Therapy Education Program at www.hemophilia.ca/gene-therapy.