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- 08/30/2017

FDA Accepts BioMarin’s Pegvaliase Biologics License Application (BLA) and grants priority review designation

Pharma Horizon

BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration  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, which we expect, when submitted, will 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

“The acceptance of this filing marks a significant milestone as we seek approval for pegvaliase as a treatment option for adults whose blood Phe levels are not adequately controlled on existing management or who are not able to control and maintain their Phe levels,” said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin. “In granting Priority Review, FDA recognizes the seriousness of PKU in adults and the associated morbidity that has substantial impact on day-to-day functioning, as well as the potential for pegvaliase to affect serious manifestations and symptoms of the disease in patients that are not adequately addressed with existing management. We look forward to working closely with FDA as they review the application to bring this important treatment to patients and offer patients an option to help with the lifelong management of PKU.”

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