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BioMarin’s Hemophilia Gene Therapy Still Works After Four Years, But Effects Tail Off

FDA Approval Expected By Late 2020

 
• By Andrew McConaghie

Latest results show a show decline in effects, but still offers freedom from injections with almost no bleeds recorded in 13 patients.

Biomarin
BioMarin expects to reach profitability in 2020, and a Roctavian approval would cap the year off

BioMarin Pharmaceutical Inc. has strengthened its case for a US Food and Drug Administration approval of its hemophilia A gene therapy (valoctocogene roxaparvovec or valrox) after new data showed it continued to work in patients four years after a one-off administration.

The Phase I/II data should help it secure an expected approval from the US regulator by the end of this year, making it the first such

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