Biomarin Submits BLA for New Phenylketonuria Therapy

PKU is a genetic disorder that causes phenylalanine hydroxylase (PAH) deficiency
PKU is a genetic disorder that causes phenylalanine hydroxylase (PAH) deficiency

BioMarin announced the submission of its Biologics License Application (BLA) to the Food and Drug Administration (FDA) for Pegvaliase. The biologic is a PEGylated recombinant phenylalanineammonia lyase enzyme product intended to reduce blood phenylalanine (Phe) levels in adults with phenylketonuria (PKU) with uncontrolled blood Phe levels.

PKU is a genetic disorder that causes phenylalanine hydroxylase (PAH) deficiency. Currently, patients with PKU are managed with a Phe-restricted diet along with low-protein modified foods and Phe-free medical foods.

The investigational drug is intended to substitute the deficient PAH enzyme with a PEGylated phenylalanine lyase enzyme to break down Phe. Clinical studies indicated that treatment with subcutaneous Pegvaliase substantially reduced blood Phe compared to placebo and led to long-term maintenance of Phe reduction for the majority of adults. Pegvaliase also demonstrated a tolerable safety profile, mostly consisting of immune-mediated responses. 

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Following the BLA submission, the FDA will conduct an initial assessment of the application and will notify the applicant of the filing decision as well as an expected Prescription Drug User Fee Act (PDUFA) date within 60 to 74 days after application receipt.

For more information call (800) 983-4587 or visit Biomarin.com