BioMarin announced the U.S. Food and Drug Administration (FDA) will require additional time to complete its review of the Biologics License Application (BLA) for its investigational therapy 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. In a notice received from the FDA, the Prescription Drug User Fee Act (PDUFA) Goal Date for pegvaliase has been extended by three months to May 28, 2018. Due to the Memorial Day weekend, the Action Goal Date will be May 25, 2018.
On Aug. 29, 2017, when the FDA accepted BioMarin's BLA and granted priority review status, the company announced that the FDA had requested additional information on Chemistry, Manufacturing and Controls (CMC), which was likely to be classified as a major amendment to the BLA and result in a three month extension of the PDUFA date. As expected, the FDA designated the receipt of this additional information as a major amendment to the application thus extending the PDUFA action date by three months.
"We are pleased with our ongoing interactions with the FDA on the pegvaliase BLA. We appreciate the FDA's ability to expedite review through priority designation, particularly for this complex disease and treatment," said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin. "We continue to work closely with the FDA and look forward to the possibility of bringing this important treatment to patients."
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.
Pegvaliase is an investigational study drug that substitutes the deficient PAH enzyme in PKU with the PEGylated version of the enzyme phenylalanine ammonia lyase, to break down Phe. It is being developed as a potential treatment for adults with inadequately controlled blood Phe levels in the study. In clinical studies, treatment with subcutaneous pegvaliase substantially reduced blood Phe compared to placebo using a randomized withdrawal study design, and led to long-term maintenance of Phe reduction in the majority of adult patients with PKU. Pegvaliase was administered using a dosing regimen that achieved a manageable safety profile, consisting primarily of immune-mediated responses, including anaphylaxis, for which robust risk management measures effective in clinical trials will be proposed.