FDA Approves New Treatment for Rare Genetic Disease

The US Food and Drug Administration has approved the subcutaneous injection Palynziq (pegvaliase-pqpz) for the treatment of adults with phenylketonuria (PKU), a rare and serious genetic disease that affects only about 1 in 10,000 to 15,000 Americans.

In PKU, the body is unable to break down phenylananine (Phe), which is commonly found in protein-containing foods and high-intensity sweeteners used in various foods and beverages.

Palynziq was approved following 2 successful clinical trials of adult patients with PKU with blood phenylalanine concentrations greater than 600 µmol/L on existing management.

In both trials, Palynziq-treated patients experienced statistically significant reductions in phenylananine concentrations from their pre-treatment baseline blood Phe concentrations.

The most commonly reported events among trial participants included injection site reactions, joint pain, hypersensitivity reactions, and headache, among others.

The most serious adverse reaction found to occur among trial participants was anaphylaxis. Because of this, Palynziq includes a boxed warning and is only available through the restricted Palynziq Risk Evaluation and Mitigation Strategy Program.

—Christina Vogt

Reference:

FDA approves a new treatment for PKU, a rare and serious genetic disease [press release]. Silver Spring, MD. US Food and Drug Administration. May 24, 2018. https://www.prnewswire.com/news-releases/fda-approves-a-new-treatment-for-pku-a-rare-and-serious-genetic-disease-300654782.html Accessed on May 25, 2018.