BioMarin Pharmaceutical Inc, a global biotechnology company, today (31 August) announced that an individual in Germany with severe haemophilia A was treated with ROCTAVIAN (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been given commercially in Europe.
In the US, ROCTAVIAN is now commercially available as Hemophilia Treatment Centers begin screening eligible individuals.
The gene therapy drug was developed from research at UCL and St Jude Children’s Research Hospital.
“Today represents an important milestone for the haemophilia 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.”
Hemophilia treatment centers (HTCs) in Germany are testing people with haemophilia 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.
“The burden of severe haemophilia 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 haemophilia A.”
Beyond 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 reimbursement.
In June, the FDA approved ROCTAVIAN for the treatment of adults with severe haemophilia 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.
ROCTAVIAN is now commercially available in the U.S. and HTCs have begun screening people with severe haemophilia A to determine eligibility. BioMarin has also seen an increasing inflow of patient consent forms and a number of executed or in-process warranty agreements that are expected to facilitate access and uptake of ROCTAVIAN at HTCs across the U.S.