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 ...

The safety profile and overall efficacy trends in adolescents were consistent with the known profile of the medicine in adults. The vast majority of adverse events (AEs) in the study were manageable, ...

If you or a loved one has phenylketonuria, you may be considering treatment options. One medicine you might hear about is Palynziq (pegvaliase-pqpz). Phenylketonuria, or PKU for short, is a rare ...

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 ...

The approval was based on data from a phase 3 open-label extension study that evaluated the long-term efficacy and safety of Palynziq in 86 adults with PKU. The Food and Drug Administration (FDA) has ...

BioMarin Pharmaceutical is continuing to slim down its pipeline, this time ending work on a preclinical phenylketonuria (PKU) drug once touted as a potential successor to its approved med Palynziq.

It's the first substitution therapy approved in Europe to target the underlying cause of PKU by helping the body to break down Phe. The European Commission has approved BioMarin’s Palynziq (pegvaliase ...

BioMarin (BMRN) Pharmaceutical announced new data characterizing the efficacy and safety of PALYNZIQ for the treatment of adolescents with phenylketonuria, PKU, which were presented at the 15th ...

SAN RAFAEL, Calif., Oct. 29, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the ...

Company plans to submit for regulatory approval later this year SAN RAFAEL, Calif., April 2, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS ...