BioMarin's hemophilia A candidate accepted for FDA priority review

21 February 2020
biomarinbig

The US Food and Drug Administration has accepted for Priority Review the Biologics License Application (BLA) for the investigational AAV5 gene therapy ValRox (valoctocogene roxaparvovec) for adults with hemophilia A submitted by California-based biotech BioMarin Pharmaceutical (Nasdaq: BMRN).

This acceptance by the FDA marks the first marketing application accepted for a gene therapy product for any type of hemophilia in the USA and, if approved, ValRox would become the first gene therapy for any type of hemophilia cleared by the US regulator.

Forecasts by analytics firm GlobalData suggest that the hemophilia market will reach a value of $9.3 billion across the eight major markets by 2028.

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