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FDA Approves BioMarin's ROCTAVIAN™ (valoctocogenoroxaparvoveque) as First Gene Therapy for Severe Hemophilia A in Adu

Friday, July 07, 2023

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) has announced that the United States Food and Drug Administration (FDA) has granted approval for ROCTAVIAN™ (valoctocogene purple parvoveque), a gene therapy designed for the treatment of severe hemophilia A in adults. This approval makes ROCTAVIAN™ the first gene therapy for severe hemophilia A to be approved in the United States, following its approval by the European Medicines Agency (EMA) in August 2022.

Hemophilia A is a genetic condition characterized by low levels of factor VIII (FVIII) activity, which can lead to increased risks of bleeding and joint damage. The current standard of care involves frequent infusions or injections of clotting factor to prevent bleeding episodes. However, ROCTAVIAN™ aims to address the underlying cause of the condition by replacing the faulty gene responsible for FVIII production. This enables individuals with severe hemophilia A to produce their own FVIII, potentially reducing the frequency of bleeding episodes.

The approval of ROCTAVIAN™ offers new hope for adults with severe hemophilia A, as it provides a single-dose treatment option that may lead to long-term control of bleeding following infusion. Dr. Steven Pipe, a professor of pediatrics and pathology at the University of Michigan and an investigator on the Phase 3 study, emphasizes the transformative potential of this gene therapy in improving the lives of individuals with severe hemophilia A.

BioMarin, a global biotechnology company focused on genetic discoveries, has a strong track record in developing innovative therapies for life-threatening genetic conditions. The manufacturing of ROCTAVIAN™ takes place at BioMarin's facility in Novato, California, which is one of the largest gene therapy manufacturing sites. This facility ensures the company's ability to meet commercial demand for ROCTAVIAN™ throughout its lifecycle.

BioMarin expresses gratitude to the clotting disorders community, as well as the patients and healthcare professionals who participated in clinical trials. The approval of ROCTAVIAN™ represents a significant milestone in advancing the field of gene therapy and brings new possibilities for the treatment of severe hemophilia A in adults.

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