Rare Disease Report

FDA Priority Review Designation Granted to BioMarin for Pegvaliase

AUGUST 30, 2017
Mathew Shanley
Yesterday, BioMarin announced that the U.S. Food and Drug Administration (FDA) accepted the company’s Biologics License Application (BLA) and granted Priority Review to pegvaliase for the treatment of adult patients with phenylketonuria (PKU).

Pegavliase is a PEGylated recombinant phenylalanine ammonia lyase enzyme product intended to reduce the levels of the amino acid phenylalanine (Phe) in adult PKU patients.

PKU is a genetic disorder in which the patient has a reduced ability to metabolize Phe. In patients, an untreated buildup of Phe. can result in cognitive issues, intellectual disability and an array of other serious health-related issues. Globally, it affects approximately 50,000 people.

The condition can be managed effectively with a Phe-restricted diet, which is characterized by low-protein modified and Phe-free foods.

Pegvaliase breaks down Phe by substituting the deficient PAH enzyme in PKU with the PEGylated version of the enzyme phenylalanine ammonia lyase. In recent clinical trials, pegvaliase was administered using a dosing regimen consisting predominantly of immune-mediated responses, including anaphylaxix, and achieved a manageable safety profile. Treatment with intravenous pegvaliase significantly reduced blood Phe reduction in most adult patients with PKU.

The priority review designation is granted to drugs that treat a critical condition, and if approved, would provide a significant improvement in safety or efficacy of the treatment, prevention, or diagnosis.

The PDUFA action date is Februrary 28, 2018. The FDA, however, has requested supplementary Chemistry, Manufacturing, and Controls (CMC) information, which, when submitted, will be classified as a major amendment and result in a 3-month extension of the PDUFA date.

If approved, pegvaliase will complement BioMarin’s other PKU therapy, Kuvan (sapropterine dihydrochloride), a drug intended to lower blood levels of Phe. in people with a certain type of PKU.

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