FDA Approves Palynziq for PKU

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.

Comment: Palynziq is available only through a restricted program called the Palynziq REMS..

Comment: BioMarin Pharmaceutical Inc announced in March 2018 that the EMA has accepted BioMarin's submission of a Marketing Authorization Application (MAA) for pegvaliase.