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Phase 3
Development Phase
0
Upcoming Catalysts
0
Historical Events
2
Regulatory Approvals
No catalyst events have been recorded for this drug yet.
Target Indication
Inborn Errors of Bile Acid Synthesis
Clinical Trial
NCT01115582Last updated: 12/4/2025
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.
Orphacol
Orphacol is indicated for the treatment of inborn errors in primary bile-acid synthesis due to 3?-hydroxy-?5-C27-steroid oxidoreductase deficiency or ?4-3-oxosteroid-5?-reductase deficiency in infants, children and adolescents aged one month to 18 years and adults.
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