BioMarin Pharmaceuticals' Biologics License Application (BLA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, has been accepted for Priority Review by the U.S. Food and Drug Administration.
Pegvaliase treats adult patients with phenylketonuria (PKU) with uncontrolled blood Phe levels by attempting to reduce the Phe levels. A decision on approval from the FDA is expected on Feb. 28, 2018.
"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," Dr. Hank Fuchs, president of worldwide research and development at BioMarin, said in a statement. "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."
Fuchs said he looks 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.