The U.S. Food and Drug Administration has approved PTC Therapeutics' oral drug to treat a rare inherited metabolic disorder ...

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

The U.S. Food and Drug Administration (FDA) has approved PTC Therapeutics (NASDAQ:PTCT) oral drug Sephience to treat ...

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 indicated to reduce blood Phe concentrations in adults with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 μmol/L on existing management.

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

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.

Palynziq is made from an enzyme, phenylalanine ammonia-lyase (PAL), found in plants, fungi and bacteria, Harding said. It replaces the enzyme that PKU patients lack, phenylalanine hydroxylase or PAH.

FRIDAY, May 25, 2018 (HealthDay News) -- Palynziq (pegvaliase-pqpz) has been approved by the U.S. Food and Drug Administration to treat phenylketonuria, commonly called PKU.

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 ...

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 ...