In Germany, the first patient with severe hemophilia A has been treated with valoctocogene roxaparvovec-rvox (Roctavian®), BioMarin Pharmaceutical announced.

“Today represents an important milestone for the hemophilia community, and for patients and physicians around the world seeking access to Roctavian,” stated Jeff Ajer, executive vice president and chief commercial officer at BioMarin.

Several hemophilia treatment centers across Germany have begun clinical evaluations and patient testing to determine if dozens of candidates are eligible to receive the one-time gene therapy. Simultaneously, final federal price negotiations are taking place.

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BioMarin Pharmaceutical’s applications seeking price and reimbursement approvals and other launch preparation activities are ongoing in France and Italy. The conditional innovation designation, granted to the novel medication in Italy, is supposed to facilitate pricing and reimbursement.

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“Bringing this therapy to all those who can benefit is critical to improve outcomes for individuals with severe hemophilia A,” said Dr. Robert Klamroth, chief physician of the Center for Hemophilia and Hemostaseology at the Vivantes Klinikum in Berlin, Germany.

“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.”

In June, the US Food and Drug Administration (FDA) approved valoctocogene roxaparvovec-rvox for the treatment of adult patients with severe hemophilia A (congenital factor VIII deficiency with activity lower than 1 IU/dL) without antibodies to adeno-associated virus serotype 5 as detected by an FDA-approved test.

The global biotechnology company is seeing an increased number of patient consent forms and a number of executed or in-process warranty agreements that are expected to facilitate access and uptake of the novel drug at hemophilia treatment centers across the United States.


BioMarin announces first person treated commercially with ROCTAVIAN® (valoctocogene roxaparvovec-rvox) for severe hemophilia A in Europe. News release. BioMarin Pharmaceutical Inc.; August 30, 2023.