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The ROCTAVIAN gene therapy has been given commercially for the first time in Europe. The European Commission (EC) granted conditional marketing authorization to ROCTAVIAN on August 24, 2022. In the U.S., ROCTAVIAN is also now commercially available with hemophilia treatment centers beginning to screen for eligible individuals.
Global biotechnology company, BioMarin Pharmaceutical Inc., announced that an individual from Germany with severe hemophilia A was treated with ROCTAVIAN® (valoctocogene roxaparvovec-rvox).
BioMarin’s executive vice president and chief commercial officer, Jeff Ajer, commented on this significant milestone for the hemophilia community and for patients and physicians seeking access to ROCTAVIAN:
“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.”
ROCTAVIAN is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without antibodies to adeno–associated virus serotype 5 (AAV5) detected by an FDA–approved test. infusion works by delivering a functional gene that is designed to enable the body to produce FVIII on its own, reducing the need for ongoing prophylaxis, as demonstrated by results reported through a 3-year study period.
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Hemophilia treatment centers (HTCs) in Germany are clinically evaluating and testing individuals with hemophilia to determine their eligibility for ROCTAVIAN. Currently, dozens of individuals are undergoing screening to determine eligibility for treatment with ROCTAVIAN with final federal price negotiations in Germany ongoing. ROCTAVIAN is now commercially available in the U.S. and HTCs have begun screening people with severe hemophilia A to determine eligibility.
BioMarin’s application for pricing 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 accommodate pricing and reimbursement.
In June, the FDA approved ROCTAVIAN for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.
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, said:
“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.”
Source: BioMarin Press Release
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