Alkaptonuria

AKU · Rare Disease · 1 drugs · 1 indications

Rare metabolic disorder where homogentisic acid accumulates in the body, depositing as dark pigment in cartilage and connective tissue (ochronosis). Over decades, joints slowly turn black and deteriorate. Like rust slowly spreading through the body's joints. ~1 in 250,000-1,000,000.
Competitive Landscape (1 drugs)
DrugCompanyMechanismModalityRouteStage
OrfadinSOBI4-hydroxyphenylpyruvate dioxygenase inhibitorSmall moleculePOAPPROVED
Indications (1)
Alkaptonuria
Orfadin APPROVED
Data from Supabase · Updated 2026-03-24