FDA approves BioMarin’s enzyme substitution therapy to treat PKU

The US Food and Drug Administration (FDA) has approved pharmaceutical company Biomarin’s Palynziq to treat phenylketonuria (PKU). This is the first enzyme substitution therapy for PKU approved by the FDA that targets the underlying cause of PKU by helping the body to reduce blood levels of phenylalanine (Phe).

PKU is a genetic order caused by a deficiency of phylalanine hydroxylase (PAH) enzyme, which is needed to reduce blood concentrations of Phe, an amino acid found in all forms of protein. High levels of Phe are toxic to the brain and can cause neurological and neuropsychiatric-related issues, such as severe intellectual disability, seizures, terrors and behavioural problems.

Palynziq is a PEGylated recombinant phenylalanine ammonia lyase enzyme which substitutes for the deficient PAH in PKU patients and breaks down the Phe reducing the blood concentration to normal levels.

FDA manging director and director of the Office of Drug Evaluation III in the Centre for Drug Evaluation and Research Julie Beitz said: "This is a novel enzyme substitution therapy that helps address a significant unmet need in PKU patients who have been unable to control their blood Phe levels with current treatment options. This new approval demonstrates our commitment to approving advancements in treatment that will give patients living with PKU different options for care."

BioMarin chief executive officer and chairman Jean-Jacques Bienaimé said: "BioMarin is thrilled to be able to offer this important new therapy to adults with PKU who are unable to control their Phe levels with existing options. The approval of Palynziq is the culmination of more than a decade of perseverance by BioMarin employees dedicated to bringing treatments to PKU adult patients.

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