EMA accepts MAA for pegvaliase to treat phenylketonuria.- BioMarin.
BioMarin Pharmaceutical Inc announced that the European Medicines Agency (EMA) has accepted BioMarin's submission of a Marketing Authorization Application (MAA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, 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.
Comment: The FDA 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 25 May, 2018.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.