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# BioMarin Rare Disease Drug Approved
- URL: https://www.fdaweb.com/biomarin-rare-disease-drug-approved/
- Published: 2018-05-25T12:00:00.000Z
- Updated: 2026-09-15T00:08:06.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5141535

FDA has approved a BioMarin Pharmaceutical NDA for Palynziq (pegvaliase-pqpz) for adults with phenylketonuria (PKU), a rare and serious genetic disease where patients are born with an inability to break down phenylalanine, present in protein-containing foods and high-intensity sweeteners used in a variety of foods and beverages. “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,” an FDA [release](https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm608835.htm?ref=fdaweb.com) says.  

Approval is based on data from two studies. Patients on therapy achieved statistically significant reductions in blood phenylalanine concentrations from their pre-treatment baseline blood concentrations, the agency says. The most common adverse events reported included injection site reactions, joint pain, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days, pruritus, nausea, dizziness, abdominal pain, throat pain, fatigue, vomiting, cough and diarrhea.