One-time gene therapy for haemophilia A gets FDA green light

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The US Food and Drug Administration (FDA) has approved the first gene therapy for adults with severe haemophilia A.

BioMarin’s Roctavian (valoctocogene roxaparvovec-rvox) has been approved 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 was first approved by the European Medicines Agency in August 2022.

“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,” said Dr Steven Pipe, Professor of Pediatrics and Pathology at the University of Michigan and an investigator in the Phase III study. “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.”

The FDA approval is based on data from the global Phase III GENEr8-1 study, the largest Phase III trial of any gene therapy in haemophilia. The 112 patients in whom six-month baseline annualised bleeding rate (ABR) was collected prospectively experienced a mean ABR reduction of 52% after receiving Roctavian (2.6 bleeds/year) compared to routine FVIII prophylaxis (5.4 bleeds/year).

These patients also reported a substantial reduction in the rate of spontaneous bleeds and joint bleeds following treatment with Roctavian compared to their baseline rate while receiving routine FVIII prophylaxis.

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