Hereditary tyrosinemia type 1

HT1 · Rare Disease · 1 drugs · 1 indications

Rare metabolic disease where the body cannot break down the amino acid tyrosine. Toxic byproducts accumulate and damage the liver, kidneys, and nervous system. Like a factory where one conveyor belt is broken, causing toxic waste to pile up. Without treatment, leads to liver failure in infancy.
Competitive Landscape (1 drugs)
DrugCompanyMechanismModalityRouteStage
OrfadinSOBI4-hydroxyphenylpyruvate dioxygenase inhibitorSmall moleculePOAPPROVED
Indications (1)
Hereditary tyrosinemia type 1
Orfadin APPROVED
Data from Supabase · Updated 2026-03-24