Palynziq Approved to Treat Phenylketonuria
May 25, 2018 – The U.S. FDA has approved Palynziq™ (pegvaliase-pqpz), manufactured by BioMarin, to treat adults with phenylketonuria (PKU) who have uncontrolled blood phenylalanine (Phe) concentrations with current treatment.
A rare but serious hereditary disorder, PKU affects an estimated 1 in 10,000 to 15,000 individuals in the United States. The condition prevents the body from properly breaking down phenylalanine, an amino acid found in many foods and sweeteners. Excess levels of phenylalanine can lead to severe brain damage.
Palynziq is a novel enzyme substitution therapy for patients who have not been able to adequately control Phe blood concentration with other available treatment options. It was shown in clinical trials to help patients reduce their Phe blood concentrations.
Due to a risk for anaphylaxis, Palynziq carries a Black Box Warning, and must be distributed through a Risk Evaluation and Mitigation Strategy (REMS) program. Prescribers, pharmacies, and patients must be enrolled in the program, and an epinephrine auto-injector must be prescribed along with Palynziq. Patients should receive the initial dose under the supervision of a healthcare provider who is prepared to manage an anaphylactic reaction. Dosage can be titrated up over time based on tolerability and therapeutic response.
BioMarin plans to launch Palynziq in June of 2018.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by U.S. mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- U.S. Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
Actual drug patent expiration dates and availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.
*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.