BioMarin Pharmaceutical announced that the U.S. Food and Drug Administration has approved a supplemental Biologics License Application for PALYNZIQ to include patients aged 12 years and older with phenylketonuria. The therapy is designed to reduce elevated blood phenylalanine concentrations in individuals with PKU, a rare inherited metabolic disorder requiring strict dietary management. The decision broadens access beyond adults, extending treatment eligibility to adolescents experiencing uncontrolled phenylalanine levels.
The approval was supported by data from the Phase 3 PEGASUS study, which evaluated safety and efficacy in adolescents with phenylalanine levels above recommended thresholds despite existing management. Participants receiving PALYNZIQ demonstrated significant reductions in blood phenylalanine over 72 weeks compared with diet alone. Clinical findings also indicated dietary flexibility improvements for some patients achieving target levels. Reported adverse reactions included injection site responses, joint pain, headache, fever, and hypersensitivity events, with most occurring during early treatment phases. BioMarin is also pursuing regulatory review in the European Union to extend access to adolescent patients.
Why it matters
Expanded approval increases therapeutic options for adolescents with PKU, potentially reducing long-term neurocognitive risks and improving disease management flexibility.
Source: BioMarin Pharmaceutical Inc.
