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 Joseph Schwartz of Leerink Partners reiterated a Buy rating on BioMarin ...

Wolfe Research projects Palynziq sales of $648 million, compared to consensus estimates of $669 million, and maintains its bullish stance on BioMarin shares despite the potential for Sephience to ...

BioMarin Pharmaceutical Inc. has advancements in phenylketonuria treatment. Click to learn how Palynziq's success could expand BMRN's market in 2025.

BioMarin Pharmaceutical Inc. announced that the phase 3 PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in ...

Based on the late-stage data, BioMarin plans to submit a regulatory filing for expanding the label of its PKU drug Palynziq in adolescents before this year's end.

PALYNZIQ® (pegvaliase-pqpz) is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe levels in adult patients with phenylketonuria who have uncontrolled blood Phe levels greater ...

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 ...

PALYNZIQ is a PEGylated enzyme therapy designed to substitute the deficient PAH enzyme in PKU patients, allowing for the breakdown of Phe. The treatment is currently approved in several regions for ...

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