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PALYNZIQ (pegvaliase-pqpz)

Phenylketonuria (PKU)

Approved/MarketedActive (Label expansion)

Key Facts

Indication

Phenylketonuria (PKU)

Phase

Approved/Marketed

Status

Active (Label expansion)

Company

BioMarin Pharmaceutical

About BioMarin Pharmaceutical

BioMarin Pharmaceutical is a mission-driven, revenue-generating biotech leader with a 25+ year track record of translating genetic insights into transformative medicines for rare diseases. The company's strategy centers on pursuing 'category-defining' therapies in areas of well-understood biology, leveraging core platforms in enzyme replacement therapy (ERT) and gene therapy. This approach has yielded a commercial portfolio generating significant revenue and a promising clinical pipeline, underpinned by global commercial and manufacturing infrastructure. BioMarin's financial strength and operational maturity support its ambition to sustainably deliver breakthrough science to patients worldwide.

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Therapeutic Areas

Rare Genetic Diseases Lysosomal Storage Disorders

Other Phenylketonuria (PKU) Drugs

Drug	Company	Phase	Status
SYNB1934	Synlogic	Phase 3	Completed; Did Not Meet Primary Endpoint
PKU Treatment	Cycle Pharmaceuticals	Commercial	Active
NB-20X	Nerai Biosciences	Pre-clinical	Active
NGGT002	NGGT	Phase 1/2 (inferred)	Active
SOM1311	SOM Biotech	Preclinical	Active
Repinatrabit	Otsuka	Open-Label Extension	Active
PER303	Perseo Pharma	Preclinical	Active
Engineered Native Bacteria Platform	Endure Biotherapeutics	Pre-clinical	Active
JNT-517	JCR Pharmaceuticals	Phase 1	Active
PTC923 (sepiapterin)	PTC Therapeutics	Phase 3	Active