FDA Expands Approval for BioMarin’s PALYNZIQ to Include Adolescents with PKU

Key Facts

1The U.S. FDA approved a supplemental Biologics License Application (sBLA) for PALYNZIQ to include pediatric patients 12 years and older with Phenylketonuria (PKU).

2PALYNZIQ is the only enzyme substitution therapy approved for the treatment of people with PKU.

3The Phase 3 PEGASUS study showed statistically significant blood phenylalanine (Phe) lowering compared to diet alone.

BioMarin Pharmaceutical Inc. (BMRN) announced that the U.S. FDA has approved its supplemental Biologics License Application (sBLA) for PALYNZIQ. The approval expands the drug's indication to include pediatric patients aged 12 years and older suffering from Phenylketonuria (PKU). PALYNZIQ remains the only approved enzyme substitution therapy designed to manage this rare genetic disorder. The FDA's decision was supported by data from the Phase 3 PEGASUS study, which demonstrated a statistically significant reduction in blood phenylalanine levels compared to dietary management alone. This label expansion is expected to significantly increase the addressable market for BioMarin's specialized therapy by reaching a younger demographic. Market analysts view this development as a positive driver for the company's long-term revenue growth within the biotechnology sector.