FDA Approves BioMarin’s Palynziq for Patients Aged 12 and Up
FDA approved supplemental BLA for Palynziq in patients aged 12+ making it sole enzyme substitution therapy for pediatric PKU. Phase 3 PEGASUS showed statistically significant mean blood phenylalanine reductions at Week 72 versus diet, and BioMarin is seeking EMA approval for EU adolescent use.
1. FDA Expands Palynziq Approval to Adolescents
The FDA granted a supplemental Biologics License Application for Palynziq, extending use to patients aged 12 and older with phenylketonuria, making it the only enzyme substitution therapy approved for pediatric PKU management.
2. Phase 3 PEGASUS Study Demonstrates Efficacy
Results from the PEGASUS trial showed a statistically significant mean reduction in blood phenylalanine levels at Week 72 versus diet control, validating the drug’s efficacy in adolescent populations.
3. EMA Submission Targets EU Adolescent Market
BioMarin has filed for marketing authorization with the European Medicines Agency to expand Palynziq use to adolescents in the EU, aiming to replicate FDA’s expanded label in European territories.
4. Stock Technicals and Analyst Outlook
Shares trade below key 20-day and 100-day SMAs and near 52-week lows, with a neutral RSI and bearish MACD; analysts maintain a Buy consensus with an average target of $88.80, with recent adjustments from Canaccord Genuity, Bernstein and Guggenheim.