Phenylketonuria

PKU · Rare Disease · 1 drugs · 1 indications

Inherited metabolic disorder caused by mutations in the PAH gene, leading to accumulation of phenylalanine causing intellectual disability if untreated.

Competitive Landscape (1 drugs)

Drug	Company	Mechanism	Modality	Route	Stage
Repinatrabit	4578	PAH Stabilizer (Phenylalanine Hydroxylase Chaperone)	Small molecule	ORAL	PHASE3

Indications (1)

PKU (Phenylketonuria)

Repinatrabit PHASE3

Upcoming Catalysts

Repinatrabit - PKU - Ph3 - Topline (NCT06971731)CLINICAL

4578Early 2027

Data from Supabase · Updated 2026-03-24