BioMarin Pharmaceutical announced 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. 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.
"The acceptance of the pegvaliase application for review by the EMA signifies a milestone in our journey to bring this important treatment to patients and families worldwide, offering a new option with the potential to alter the course of lifelong PKU management," said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin. "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."
Pegvaliase is an investigational study drug that substitutes the deficient PAH enzyme in PKU with the PEGylated version of the enzyme phenylalanine ammonia lyase, to break down Phe. It is being developed as a potential treatment for adults with inadequately controlled blood Phe levels in the study. In clinical studies, treatment with subcutaneous pegvaliase substantially reduced blood Phe compared to placebo using a randomized withdrawal study design, and led to long-term maintenance of Phe reduction in the majority of adult patients with PKU. Pegvaliase was administered using a dosing regimen that achieved a manageable safety profile, consisting primarily of immune-mediated responses, including anaphylaxis, for which robust risk management measures effective in clinical trials will be proposed.