European Medicines Agency (EMA) has accepted BioMarin’s submission of a Marketing Authorization Application (MAA) for pegvaliase for the treatment of adults with phenylketonuria (PKU) who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options including sapropterin.
The U.S. Food and Drug Administration accepted the Biologics License Application (BLA) for pegvaliase and granted priority review status in August 2017, with the Prescription Drug User Fee Act (PDUFA) Action Goal Date of May 25, 2018.
Hank Fuchs, President Worldwide Research and Development at BioMarin, said: “For more than 10 years, we have been committed to advancing the development of therapies for the PKU community, and we look forward to working with European regulatory authorities on the pegvaliase application.”