EMA delays review of BioMarin hemophilia A candidate

10 September 2020

In a Securities Exchange Commission filing yesterday, California-based biotech BioMarin Pharmaceutical (Nasdaq: BMRN) revealed it has received the European Medicines Agency’s Joint Assessment Report related to the EMA’s ongoing review of the company’s Marketing Authorization Application for ValRox (valoctocogene roxaparvovec) for severe hemophilia A.

The Report requests that BioMarin submits to the EMA the full 52-week results from the 134 patients in the ongoing Phase III study of valoctocogene roxaparvovec with the 6e13 vg/kg dose.

The company expects the last patient will reach 52 weeks of follow-up in November 2020, and BioMarin is working with the EMA to enable a potential submission of the requested data by the end of the first quarter of 2021.

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