BioMarin Pharmaceutical has announced that a patient in Germany with severe haemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), signifying the first time that the gene therapy has been given commercially in Europe.
Jeff Ajer, BioMarin’s executive vice president and chief commercial officer, said the announcement “represents an important milestone” for the haemophilia community, as well as for patients and physicians around the world seeking access to the therapy.
Haemophilia treatment centres in Germany are currently testing patients to determine treatment eligibility, the company said, adding that final federal price negotiations in the country are ongoing.
People living with haemophilia A lack sufficient functioning factor VIII protein to help their blood clot, putting them at risk of painful and potentially life-threatening bleeds.
Patients with the most severe form of the disease, which make up around 50% of the haemophilia A population, are currently treated with intravenous factor VIII infusions two to three times per week, or a bispecific monoclonal antibody one to four times per month.
Despite these prophylactic regimens, many patients continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage.
BioMarin’s Roctavian, which is administered as a one-time infusion, delivers a functional gene that is designed to enable the body to produce factor VIII on its own without the need for continued haemophilia prophylaxis, relieving patients of their treatment burden.
The therapy was granted conditional marketing authorisation by the European Commission in August 2022 to treat adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5.
Beyond Germany, the company’s applications seeking price and reimbursement approvals and other launch preparation activities for Roctavian continue to progress in France and Italy.
“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,” said Dr Robert Klamroth, a treating physician and chief physician of the Center for Hemophilia and Hemostaseology at the Vivantes Klinikum in Germany. “Bringing this therapy to all those who can benefit is critical to improve outcomes for individuals with severe haemophilia A.”
The announcement comes just a month after the US Food and Drug Administration approved Roctavian for adults with severe haemophilia A.