BioMarin Pharmaceutical (BMRN) Receives FDA Approval for Expanded Use of PALYNZIQ in Adolescents

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best get-rich-quick stocks to buy according to hedge funds. On February 27, the US FDA approved BioMarin’s supplemental Biologics License Application for PALYNZIQ, expanding its use to include adolescents aged 12 and older with phenylketonuria/PKU. This medication remains the only approved enzyme substitution therapy designed to reduce blood phenylalanine/Phe concentrations in patients with this rare genetic condition.

The approval is particularly significant for teenagers, as the restrictive dietary management required for PKU often becomes difficult to maintain during adolescence, leading to potential neurocognitive issues if blood Phe levels remain uncontrolled. Data from the Phase 3 PEGASUS study supported this decision, demonstrating that PALYNZIQ achieved statistically significant reductions in blood Phe levels compared to diet alone.

BioMarin Pharmaceutical (BMRN) Receives FDA Approval for Expanded Use of PALYNZIQ in Adolescents

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The safety profile for adolescents was found to be consistent with previous studies in adults, with common side effects including injection site reactions, joint pain, and headaches. Owing to the risk of anaphylaxis, the therapy is administered through a restricted program under a Risk Evaluation and Mitigation Strategy/REMS. BioMarin Pharmaceutical Inc. is currently pursuing a similar expansion of the drug’s label with the European Medicines Agency to reach the adolescent population in the European Union.

BioMarin Pharmaceutical Inc. is a biotechnology company that develops and commercializes therapies for life-threatening rare diseases and medical conditions in the US, Europe, Latin America, the Middle East, the Asia Pacific, and internationally.

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