BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) released topline data from the Phase 3 PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) compared to diet alone in 55 adolescents aged 12-17 with phenylketonuria (PKU).

PKU is a rare, inherited metabolic disorder that prevents the body from breaking down the amino acid phenylalanine. Untreated phenylketonuria can lead to brain damage, intellectual disabilities, behavioral symptoms or seizures.

The primary endpoints are the changes in blood Phe concentration and characterization of the safety profile in adolescents. Secondary endpoints include changes in total dietary protein intake and pharmacokinetics. 

Also Read: Why Is BioMarin Stock Trading Higher On Thursday?

The study met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone.

Safety results were consistent with the known profile of the medicine.

Palynziq is the first and only enzyme therapy approved to treat adults with PKU.

Detailed results from the PEGASUS study will be presented at an upcoming medical meeting and submitted to global health authorities later this year to request a label expansion for Palynziq to include adolescents.

Palynziq substitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with a PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe. Palynziq is administered using a dosing regimen designed to facilitate tolerability.

Palynziq is approved to reduce blood Phe concentrations for adults in the U.S., for people 16 and older in the EU, Canada, and Brazil, and for people 15 and older in Japan with PKU who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.

Price Action: BMRN stock is up 0.29% at $68.14 at the last check on Wednesday.

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This article BioMarin Seeks Label Expansion For Palynziq After Positive Phase 3 Results In Adolescent Patients originally appeared on Benzinga.com

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