FDA approves BioMarin’s Roctavian for adults with severe haemophilia A

Betsy Goodfellow | June 30, 2023 | News story | Medical Communications BioMarin, FDA, Haematology, Roctavian, haemophilia 

Global biotechnology company BioMarin Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has approved Roctavian (valoctocogene roxaparvocec-rvox) gene therapy for the treatment of adult patients 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.

The drug is a one-time, single-dose infusion and is now the first gene therapy for severe haemophilia A to be approved in the US. Roctavian was also the first to be approved by the European Medicines Agency in August 2022.

Dr Steven Pipe, professor of paediatrics and pathology at the University of Michigan, US, and an investigator on the phase 3 trial, commented: “Adults with severe haemophilia A face a lifelong burden, with frequent infusions and a high risk of health complications, including uncontrolled bleeding and irreversible joint damage. The approval of Roctavian, as the first gene therapy for severe haemophilia A, has the potential to transform the way we treat adults based on years of bleed control following a single, one-time infusion.”

Jean-Jacques Beinaimé, chairman and CEO of BioMarin, added: “Today’s approval of Roctavian builds on BioMarin’s proven track record of advancing treatments that target the underlying cause of life-threatening genetic conditions, which has produced eight best- or first-in-class commercial therapies. We are proud to now offer adults with severe haemophilia A, a one-time, single-dose treatment option. We are especially grateful to the bleeding disorders community for its support of this program, and to all the patients and healthcare providers who participated in our clinical trials.”

Betsy Goodfellow

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