Investors unimpressed by BioMarin data

Phase III findings on BioMarin Pharmaceutical’s (Nasdaq: BMRN) valoctocogene roxaparvovec were announced on Tuesday, leading the US biotech’s share value to fall by 5% by the close of trading.

The gene therapy is being investigated in severe hemophilia A and has breakthrough therapy and PRIME designations from the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) respectively.

As of May 28, eight patients in the 20-patient cohort of the GENEr8-1 study achieved Factor VIII levels of 40 international units per deciliter, or more, at 23 to 26 weeks, meeting the pre-specified criteria for Factor VIII activity levels that was set for regulators submissions in the USA and Europe.