FDA accepts for Priority Review the BLA for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product to treat phenylketonuria (PKU).-BioMarin

BioMarin Pharmaceutical Inc. announced that the FDA has accepted for Priority Review the Biologics License Application (BLA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with phenylketonuria (PKU) who have uncontrolled blood Phe levels on existing management.

The FDA has granted priority review designation to pegvaliase, which is granted to drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. The PDUFA action date is February 28, 2018. However, the FDA has requested additional Chemistry, Manufacturing, and Controls (CMC) information, is likely, when submitted, to be classified as a major amendment and result in a three month extension of the PDUFA date. The Agency has not informed the company whether an advisory committee meeting to discuss the application will be needed.