Subject: Cholbam SGM 2119-A P2025

Drug

CHOLBAM  (cholic acid)

Policy:

Indications

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.

FDA-approved Indications¹

Cholbam is indicated for:

• Treatment of bile acid synthesis disorders due to single enzyme defects (SEDs).
• Adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat-soluble vitamin absorption.

Limitations of Use

The safety and effectiveness of Cholbam on extrahepatic manifestations of bile acid synthesis disorders due to SEDs or PDs including Zellweger spectrum disorders have not been established.

All other indications are considered experimental/investigational and not medically necessary.

Documentation

Submission of the following information is necessary to initiate the prior authorization review:

• Initial requests:
  • Mass spectrometry, enzyme assay, biochemical testing results, or genetic testing results confirming diagnosis; and
  • Lab test results documenting baseline liver function (i.e., transaminases, bilirubin, presence of cholestasis).
• Continuation of therapy requests: lab results documenting an improvement in liver function (i.e., reduced transaminases, reduced bilirubin, no evidence of cholestasis on liver biopsy).

Prescriber Specialties

This medication must be prescribed by or in consultation with a physician who specializes in the treatment of enzyme or metabolic disorders.

Coverage Criteria

Bile Acid Synthesis Disorders Due to Single Enzyme Defects (SEDs)^1-3

Authorization of 6 months may be granted for treatment of bile acid synthesis disorders due to single enzyme defects when both of the following criteria are met:

• The diagnosis is confirmed by mass spectrometry or other biochemical testing, genetic testing, or enzyme assay.
• The member has liver dysfunction (i.e., elevated transaminases, elevated bilirubin, presence of cholestasis) at baseline.

Peroxisomal Disorders (PDs) Including Zellweger Spectrum Disorders^1,4

Authorization of 6 months may be granted for adjunctive treatment of peroxisomal disorders when both of the following criteria are met:

• The diagnosis is confirmed by mass spectrometry or other biochemical testing, or by genetic testing.
• The member exhibits manifestations of liver disease (i.e., elevated transaminases, elevated bilirubin, presence of cholestasis).

Continuation of Therapy

Authorization of 12 months may be granted to members with an indication listed in the coverage criteria section who are currently receiving the requested medication through a paid pharmacy or medical benefit, and who have achieved and maintained improvement in liver function from baseline (i.e., reduced transaminases, reduced bilirubin, no evidence of cholestasis on liver biopsy).

Place of Service:

Outpatient

The above policy is based on the following references:

1. Cholbam [package insert]. San Diego, CA: Manchester Pharmaceuticals, Inc. a wholly owned subsidiary of Travere Therapeutics, Inc.; March 2023.
2. Gonzales E, Gerhardt MF, Fabre M et al. Oral cholic acid for hereditary defects of primary bile acid synthesis: a safe and effective long-term therapy. Gastroenterology. 2009;137:1310-1320.
3. Heubi J, Setchell KDR, Bove KE. Inborn errors of bile acid metabolism. Seminars Liver Dis. 2007;27:282-294.
4. Poll-The BT, Gartner J. Clinical diagnosis, biochemical findings and MRI spectrum of peroxisomal disorders. Biochim Biophys Acta. 2012;1822:1421-1429.

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

May 18, 2025