Posted by Michael Wonder on 29 Mar 2018
EMA accepts BioMarin's marketing application for pegvaliase MAA for treatment of phenylketonuria
28 March 2018 - BioMarin Pharmaceutical announced today that the EMA has accepted BioMarin's submission of a marketing authorisation application for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, for the treatment of adults with phenylketonuria who have inadequate blood phenylalanine control despite prior management with available treatment options including sapropterin.
"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."
