FDA OKs New Treatment for Phenylketonuria

FDA officials have approved pegvaliase-pqpz (Palynziq, BioMarin Pharmaceutical) for adults with a rare and serious genetic disease, phenylketonuria (PKU). Patients with PKU are born with an inability to break down phenylalanine (Phe), an amino acid present in high-intensity sweeteners and protein-containing foods used in a variety of beverages and foods. Pegvaliase-pqpz is indicated for adult PKU patients who have uncontrolled blood Phe concentrations on current treatment.

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“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,” Julie Beitz, MD, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research, said in a statement.

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“This new approval demonstrates our commitment to approving advancements in treatment that will give patients living with PKU different options for care.”

PKU affects about 1 in 10,000 to 15,000 people in the United States. If untreated, PKU can cause chronic intellectual, neurodevelopmental, and psychiatric disabilities. Lifelong restriction of Phe intake through the diet is needed to prevent buildup of Phe in the body, which can cause long-term damage to the central nervous system.

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