BioMarin Pharmaceutical Inc. (BMRN) said that it received standard approval from the U.S. Food and Drug Administration for Palynziq (pegvaliase-pqpz) Injection to reduce blood phenylalanine or Phe concentrations in adult patients with phenylketonuria or PKU, who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.
Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe. Palynziq is BioMarin’s second approved treatment for this important condition.
PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. PKU affects approximately 1 in 12,500 live births in the United States each year. PKU is marked by an inability to break down Phe, an amino acid that is found in all forms of protein. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychiatric-related issues, impacting the way a person thinks, feels, and acts.
Due to the seriousness of these symptoms, infants are screened at birth to ensure that they are diagnosed early and treated to avoid intellectual disability and other complications. Patients living with PKU require life-long management, including adherence to a challenging and severely restrictive daily diet of medical foods and formula that avoids the ingestion of Phe that is present in most foods.
The approval of Palynziq in the United States marks an important milestone for adults living with PKU who will now have access to an effective new treatment option for controlling their blood Phe.
The approval of Palynziq comes during National PKU Awareness Month. During National PKU Awareness Month, local patient organizations are encouraged to organize events to spread PKU awareness and raise funds for academic research.
Palynziq is expected to be available in the United States by the end of June, and BioMarin will begin the promotion of Palynziq immediately. Palynziq is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Palynziq REMS.
BioMarin is also committed to bringing Palynziq to adult PKU patients outside of the United States. In March 2018, the European Medicines Agency accepted BioMarin’s submission of a Marketing Authorization Application for Palynziq.