BioMarin submits BLA for PKU candidate

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) submitted a BLA to FDA for pegvaliase (BMN 165) to treat phenylketonuria. The candidate is a pegylated recombinant phenylalanine ammonia lyase (PAL) enzyme.

In March 2016, Biomarin said pegvaliase met the primary endpoint of change in blood phenylalanine in the Phase III PRISM-2 trial to treat PKU, but did not lead to significant improvements on key secondary endpoints. At BioMarin's end-of-Phase II meeting, FDA had said the phenylalanine endpoint could substantiate an accelerated approval, but that the company would need to show neurocognitive benefit to obtain full approval, according to President of Worldwide R&D Henry Fuchs (see BioCentury Extra, March 21, 2016)...