BioMarin's haemophilia gene therapy set for European debut after regulatory nod

 The European Union's drug regulator on Friday recommended conditional approval to BioMarin Pharmaceutical's treatment for haemophilia A, making it the first gene therapy for the bleeding disorder.

If approved by the European Commission, it would pave the way for a rare debut for Roctavian, also known as valoctocogene roxaparvovec, in Europe even before a nod from the U.S. regulator. An approval is usually granted following a recommendation from the European Medicines Agency (EMA).

Shares of U.S.-listed BioMarin rose nearly 2% in premarket trading as the EMA nod ends a long-drawn journey for the therapy whose application for EU approval had to be resubmitted in June last year after it was withdrawn in November 2020.

Gene therapies are typically expensive. The company did not respond to a request for comment on the pricing of the drug.

Stifel analyst Paul Matteis called the approval a "big landmark" and said it raises the prospects of a similar move by the U.S. drugs regulator, which had rejected the therapy in August 2020 citing the need for two-year data.

The EMA said its approval now is based on data from a global late-stage study of 134 male patients with severe haemophilia A, which showed an 85% reduction in bleeding rates two years after administration. (https://reut.rs/3HPJgWO)

The company now plans to resubmit its application to the U.S. Food and Drug Administration in September end. It had delayed an earlier plan to apply in June as it had to provide additional information.

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