FDA Approves Palynziq for Phenylketonuria
FRIDAY, May 25, 2018 -- Palynziq (pegvaliase-pqpz) has been approved by the U.S. Food and Drug Administration to treat phenylketonuria, or PKU.
Palynziq is a novel enzyme therapy for adult patients with PKU with uncontrolled blood phenylalanine (Phe) concentrations despite treatment. The drug's safety and effectiveness were evaluated in clinical studies involving patients with high concentrations of blood Phe. Statistically significant reductions in blood Phe concentrations (from pre-treatment baseline levels) were seen in patients treated with Palynziq.
The most common side effects of the enzyme substitution therapy were injection-site reactions, hypersensitivity reactions, joint pain, headache pruritus, nausea, dizziness, and abdominal pain. The drug's label warns of a less-common risk of life-threatening anaphylaxis, which the agency said most often occurred when the dosage was increased during the first year of treatment.
"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, M.D., director of the Office of Drug Evaluation III in the FDA's Center for Drug Evaluation and Research, said in a statement. "This new approval demonstrates our commitment to approving advancements in treatment that will give patients living with PKU different options for care."
Palynziq is produced by BioMarin Pharmaceutical, based in Novato, Calif.
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Posted: May 2018