FDA Approves Novel Enzyme Therapy for Rare Genetic Disease

The FDA approved Biomarin’s novel enzyme Palynziq (pegvaliase-pqpz) for treatment of the rare genetic disease phenylketonuria (PKU), indicated for treatment of adult PKU patients with uncontrolled phenylalanine.

The FDA based its clearance on two clinical trials of adult patients with PKU. The illness affects about 1 in 10,000 to 1 in 15,000 individuals in the United States.

The product includes a Boxed Warning for the risk of anaphylaxis and it is only available under a Risk Evaluation and Mitigation Strategy.