Phase 3
Development Phase
0
Upcoming Catalysts
0
Historical Events
2
Regulatory Approvals
No Catalysts Found
No catalyst events have been recorded for this drug yet.
1 competitor in Inborn Errors of Bile Acid Synthesis
View Full LandscapeTarget Indication
Inborn Errors of Bile Acid Synthesis
Clinical Trial
NCT01115582Last updated: 12/4/2025
No patent data available. Pre-approval drugs may not have Orange Book listings.
Kolbam
Cholic Acid FGK is indicated for the treatment of inborn errors of primary bile acid synthesis, in infants from one month of age for continuous lifelong treatment through adulthood, encompassing the following single enzyme defects: sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency; 2- (or alpha-) methylacyl-CoA racemase (AMACR) deficiency; cholesterol 7 alpha-hydroxylase (CYP7A1) deficiency.
View on EMAOrphacol
Orphacol is indicated for the treatment of inborn errors in primary bile acid synthesis due to3β-Hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-Oxosteroid-5β-reductase deficiency ininfants, children and adolescents aged 1 month to 18 years and adults.
View on EMA