BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the most profitable biotech stocks to invest in now. On July 23, analyst ...

SAN RAFAEL, Calif., April 2, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary ...

Palynziq is BioMarin's second approved treatment for this important condition. PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain.

The FDA has approved an increased maximum dose for Palynziq® (pegvaliase-pqpz) in the treatment of adults with phenylketonuria who have uncontrolled Phe concentrations greater than 600μmol/L on ...

Palynziq is administered using a dosing regimen designed to facilitate tolerability; Palynziq's safety profile consists primarily of immune-mediated responses, including anaphylaxis, for which ...

Palynziq is the first enzyme therapy approved for the treatment of PKU in the United States. The drug will complement sales of Kuvan, also approved for PKU, but has lost exclusivity in the country.

BioMarin Pharmaceutical Inc. BMRN announced that the FDA has approved its supplemental biologics license application (sBLA), seeking to increase the maximum allowable dose of Palynziq (pegvaliase ...

Pegvaliase [Palynziq]. Diet only [Control arm]. One important thing to note is that this particular open-label study had the control arm patients have a "diet only" option to be done for 72 weeks.

The safety and efficacy of Palynziq were studied in two clinical trials in adult patients with PKU with blood phenylalanine concentrations greater than 600 µmol/L on existing management.

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically ...