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PTC923 (sepiapterin)

Phenylketonuria (PKU)

Phase 3Active

Key Facts

Indication

Phenylketonuria (PKU)

Phase

Phase 3

Status

Active

Company

PTC Therapeutics

About PTC Therapeutics

PTC Therapeutics is a patient-centric, commercial-stage biopharma focused on discovering, developing, and commercializing clinically differentiated medicines for rare diseases. The company has achieved multiple regulatory firsts, including the first global treatment for Duchenne muscular dystrophy (Translarna), the first brain-infused gene therapy (Upstaza), and the first small molecule splicing modifier for spinal muscular atrophy (Evrysdi). Its strategy leverages two proprietary scientific platforms—Splicing and Inflammation & Ferroptosis—to build a diversified pipeline, supported by a global commercial infrastructure aimed at sustainable growth and expanding patient access.

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Therapeutic Areas

Neuromuscular Disorders Metabolic Disorders Neurodegenerative Diseases Genetic 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
PALYNZIQ (pegvaliase-pqpz)	BioMarin Pharmaceutical	Approved/Marketed	Active (Label expansion)