Product Launchenzyme substitutionrare diseasesbiomarinfda approval
BioMarin Expands PALYNZIQ Use To Adolescents
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6.6
BioMarin announced that on February 27 the US FDA approved a supplemental Biologics License Application expanding PALYNZIQ use to adolescents aged 12 and older with phenylketonuria (PKU). The approval, supported by Phase 3 PEGASUS data showing statistically significant reductions in blood phenylalanine, retains a consistent safety profile and is subject to a REMS program; BioMarin is pursuing a similar EU label expansion.
Scoring Rationale
Official FDA approval provides verifiable clinical and commercial advancement, but impact remains niche to the PKU patient population.
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