Shots:
- The US FDA has accepted sBLA & granted priority review to Palynziq for the treatment of adolescents (12-17yrs.) with phenylketonuria (PDUFA: Feb 28, 2026)
- sBLA was supported by the P-III (PEGASUS) trial comparing Palynziq to diet alone in 55 adolescents with phenylketonuria, which showed reduced blood phenylalanine (Phe); results were presented at ICIEM’25
- Palynziq uses a PEGylated phenylalanine ammonia lyase enzyme to replace deficient PAH in PKU, & the company aims to share data with the EMA to expand treatment eligibility to adolescents (≥12yrs.) in the EU
Ref: BioMarin | Image: BioMarin | Press Release
Related News:- BioMarin Pharmaceutical to Acquire Inozyme Pharma for ~$270M, Strengthening its Enzyme Therapies Portfolio
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