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Cholic Acid

Generic Name
Cholic Acid
Brand Names
Cholbam, Orphacol
Drug Type
Small Molecule
Chemical Formula
C24H40O5
CAS Number
81-25-4
Unique Ingredient Identifier
G1JO7801AE
Background

A major primary bile acid produced in the liver and usually conjugated with glycine or taurine. It facilitates fat absorption and cholesterol excretion. [PubChem]

Cholic acid, formulated as Cholbam capsules, is approved by the United States Food and Drug Administration as a treatment for children and adults with bile acid synthesis disorders due to single enzyme defects, and for peroxisomal disorders (such as Zellweger syndrome).

Indication

Oral cholic acid is indicated for: treatment of bile acid synthesis disorders due to single enzyme defects; and as adjunctive treatment of peroxisomal disorders including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat soluble vitamin absorption.

Associated Conditions
Inborn Errors of Bile Acid Synthesis, Zellweger Spectrum Disorder, Peroxisomal disorders (PDs)

FDA Approves Tablet Formulation of LIVMARLI for Rare Pediatric Liver Diseases

• Mirum Pharmaceuticals' LIVMARLI (maralixibat) has received FDA approval for a new tablet formulation to treat cholestatic pruritus in patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis. • The tablet version provides a convenient one-tablet per dose option for older patients, while maintaining the liquid formulation for younger patients, enhancing treatment flexibility for physicians and patients. • LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is now approved in over 40 countries for ALGS and 30 countries for PFIC, with the new tablet formulation expected to be available in June 2025.

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis: Mirum's Chenodiol Tablets

• The FDA has granted historic approval to chenodiol tablets (Ctexli) as the first-ever treatment for cerebrotendinous xanthomatosis, a rare genetic lipid storage disease affecting multiple organ systems. • Phase 3 RESTORE study demonstrated chenodiol's efficacy with a statistically significant 20-fold reduction in bile alcohols compared to placebo (P<0.0001) in CTX patients. • The treatment, administered as 250mg tablets three times daily, comes with monitoring requirements for liver toxicity and showed common side effects including diarrhea and headache.

Volixibat Receives FDA Breakthrough Therapy Designation for Cholestatic Pruritus in Primary Biliary Cholangitis

• The FDA granted Breakthrough Therapy Designation to volixibat for treating cholestatic pruritus in patients with primary biliary cholangitis (PBC). • This designation is based on positive interim results from the Phase 2b VANTAGE study, showcasing statistically significant pruritus improvement versus placebo. • Volixibat, an oral ileal bile acid transporter (IBAT) inhibitor, could expedite development and review for this burdensome symptom of PBC. • Enrollment in the confirmatory portion of the VANTAGE study is ongoing, with completion expected in 2026, marking a significant step forward.
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