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 The leading web portal for pharmacy resources, news, education and careers November 20, 2017
Pharmacy Choice - Pharmaceutical News - FDA Accepts BioMarin's Pegvaliase Biologics License Application BLA and Grants Priority Review Designation - November 20, 2017

Pharmacy News Article

 9/13/17 - FDA Accepts BioMarin's Pegvaliase Biologics License Application BLA and Grants Priority Review Designation

By a News Reporter-Staff News Editor at Biotech Week BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced that the U.S. Food and Drug Administration (FDA) 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 (see also Pharmaceutical Companies).

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."

Keywords for this news article include: Biotechnology, BioMarin Pharmaceutical Inc., Pharmaceutical Companies, Biologics, FDA Actions, Bioengineering, Genetic Engineering, Genetically-Engineered Proteins, Government Agencies Offices and Entities.

Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2017, NewsRx LLC

(c) 2017 NewsRx LLC

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