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 anticipated to launch by the end of June 2018 as 2.5mg/0.5 mL, 10mg/0.5 mL, and 20mg/mL strength single-dose prefilled syringes.

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.

The Palynziq drug, developed by BioMarin Pharmaceutical Inc., is an injectible shot of an enzyme similar to phenylalanine that the body can break down more easily, Sanchez-Valle said.

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 ...