BioMarin Wins FDA Approval for Palynziq in Patients Aged 12+ With PKU
BioMarin secured FDA approval for its supplemental BLA of Palynziq as the first enzyme substitution therapy for PKU in patients aged 12 and older, based on Phase 3 PEGASUS data. The trial showed significant mean blood phenylalanine reductions at Week 72 versus diet alone, and BioMarin is pursuing EMA approval.
1. FDA Approval Expands Pediatric Label
The FDA approved BioMarin's supplemental BLA for Palynziq, expanding its indication to adolescents aged 12 and older with PKU. This approval designates Palynziq as the sole enzyme substitution therapy for managing phenylalanine levels in this age group.
2. Phase 3 PEGASUS Demonstrates Efficacy
The PEGASUS trial showed that Palynziq patients achieved significant mean reductions from baseline in blood phenylalanine at Week 72 compared to diet-only controls. These data underpin the expanded label and support future EU filings.
3. Market Potential and Regulatory Pathway
With this approval, BioMarin can target the pediatric PKU segment and is pursuing a parallel submission to the European Medicines Agency for similar pediatric expansion. Analysts currently maintain a Buy consensus with an average target of $88.80, reflecting confidence in growth prospects.
4. Stock Technicals Indicate Mixed Momentum
Shares trade approximately 4.5% below their 20-day SMA and 8.2% below the 100-day SMA, closer to 52-week lows after a 19% annual decline. The RSI sits at 50.00, while the MACD at -0.10 under its 0.05 signal line signals bearish pressure.