FDA approves new PKU treatment Palynziq
The Food and Drug Administration has approved BioMarin’s Palynziq (pegvaliase-pqpz), a treatment for the rare genetic disease phenylketonuria, or PKU. Patients with PKU are unable to break down the amino acid phenylalanine, or Phe, which is present in protein-containing foods and high-intensity sweeteners. Palynziq is indicated for patients with PKU who have uncontrolled blood Phe concentrations on their current 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," said Julie Beitz, director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research. "This new approval demonstrates our commitment to approving advancements in treatment that will give patients living with PKU different options for care."
PKU affects roughly 1-in-10,000 to 15,000 people in the United States, and can cause chronic intellectual, neurodevelopmental and psychiatric disabilities. It also requires restriction of phenylalanine intake, which can damage the central nervous.
“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,” said Jean-Jacques Bienaimé, chairman and CEO of BioMarin.
The company said it expects to make Palynziq available by the end of June, with plans to promote the product immediately. The drug will only be avaialble through a Risk Evaluation and Mitigation Strategy called the Palynziq REMS.
"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," said Julie Beitz, director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research. "This new approval demonstrates our commitment to approving advancements in treatment that will give patients living with PKU different options for care."
PKU affects roughly 1-in-10,000 to 15,000 people in the United States, and can cause chronic intellectual, neurodevelopmental and psychiatric disabilities. It also requires restriction of phenylalanine intake, which can damage the central nervous.
“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,” said Jean-Jacques Bienaimé, chairman and CEO of BioMarin.
The company said it expects to make Palynziq available by the end of June, with plans to promote the product immediately. The drug will only be avaialble through a Risk Evaluation and Mitigation Strategy called the Palynziq REMS.