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🏅 FDA Orphan Designation

Palynziq

PEGVALIASE-PQPZ

Manufacturer: BioMarin Pharmaceutical Inc.

Indicated for:
Maternal phenylketonuria syndromeOrphanPhenylketonuriaOrphan
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Black Box Warning (Boxed Warning)

A Boxed Warning is the FDA's strongest safety warning — it indicates serious or life-threatening risks identified during clinical trials or post-market surveillance. It does not mean the medication is unsafe for all patients; your prescriber weighs these risks against the benefits for your specific situation. Source: FDA-approved prescribing information.

View full prescribing information on DailyMed (NLM) ↗

FDA-Approved Indications (2)

to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than

PhenylketonuriaOrphan Designation

1 INDICATIONS AND USAGE PALYNZIQ is indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled bl

Indications & Usage

1 INDICATIONS AND USAGE PALYNZIQ is indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. PALYNZIQ is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. ( 1 )

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Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.