PTC Therapeutics Could Challenge BioMarin For Metabolic Disorder Treatment

On Saturday, BioMarin Pharmaceutical Inc. BMRN released new Phase 3 PEGASUS study results on Saturday.The data show that its therapy Palynziq (pegvaliase-pqpz) significantly reduced blood phenylalanine (Phe) levels in adolescents with phenylketonuria (PKU), a rare metabolic disorder.The trial enrolled 55 patients aged 12–17, with 36 receiving Palynziq and 19 continuing on diet alone. After 72 weeks, 45% of Palynziq patients achieved at least a 50% reduction in Phe from baseline, many reaching guideline-reco ...