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BioMarin Announces First Person Treated Commercially with ROCTAVIAN® (valoctocogene roxaparvovec-rvox) for Severe Hemophilia A in Europe
First Person Treated in Germany; Reimbursement Discussions on Track in France and Italy In the U.S., ROCTAVIAN Now Commercially Available and Hemophilia
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"First Person Treated in Germany; Reimbursement Discussions on Track in France and Italy\nIn the U.S., ROCTAVIAN Now Commercially Available and Hemophilia Treatment Centers Have Begun Screening Eligible Individuals\nSAN RAFAEL, Calif., Aug. 30, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that an individual in Germany with severe hemophilia A was treated with ROCTAVIAN® (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been given commercially in Europe.\n\n \n \n \n \n \n \n\n \n\"Today represents an important milestone for the hemophilia community, and for patients and physicians around the world seeking access to ROCTAVIAN,\" said Jeff Ajer, executive vice president and chief commercial officer at BioMarin. \"We look forward to more people gaining access to ROCTAVIAN in the rest of Europe, as well as the United States, where ROCTAVIAN recently received FDA approval.\"\nHemophilia treatment centers (HTCs) in Germany are testing people with hemophilia to determine treatment eligibility for the one-time gene therapy. Before beginning treatment, individuals are evaluated clinically and undergo testing. Currently, dozens of individuals are undergoing screening to determine eligibility for treatment with ROCTAVIAN. Final federal price negotiations in Germany are also ongoing. \n\"The burden of severe hemophilia A for people who are living with the condition is substantial, and there remains a significant unmet need for effective treatments that do not require chronic therapy. This one-time infusion represents an important milestone, offering new hope and potential, for eligible individuals in Germany,\" said PD Dr. med. Robert Klamroth, a treating physician and chief physician of the Center for Hemophilia and Hemostaseology at the Vivantes Klinikum in Berlin, Germany. \"Bringing this therapy to all those who can benefit is critical to improve outcomes for individuals with severe hemophilia A.\"\nBeyond Germany, the company's applications seeking price and reimbursement approvals and other launch preparation activities continue to progress in France and Italy. In Italy, ROCTAVIAN was awarded conditional innovation designation, which is expected to facilitate pricing and reimbu...