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BioMarin Pharmaceutical (BMRN) Receives FDA Approval for Expanded Use of PALYNZIQ in Adolescents [Yahoo! Finance]

BioMarin Pharmaceutical Inc. (BMRN) 
Last biomarin pharmaceutical inc. earnings: 4/29 04:01 pm Check Earnings Report
US:NASDAQ Investor Relations: investors.biomarin.com
Company Research Source: Yahoo! Finance
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 Jirsak / shutterstock.com The safety profile for adolescents was found to be consistent with previous Show less Read more
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