Subject: Cholbam 2119-A SGM P2024
Policy:
I. 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 Indications
Cholbam is indicated for:
1. Treatment of bile acid synthesis disorders due to single enzyme defects (SEDs).
2. 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.
Limitation 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.
II. DOCUMENTATION
Submission of the following information is necessary to initiate the prior authorization review:
A. Initial requests:
1. Mass spectrometry, enzyme assay, biochemical testing results, or genetic testing results
confirming diagnosis; and
2. Lab test results documenting baseline liver function (i.e., transaminases, bilirubin,
presence of cholestasis).
B. 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).
III. CRITERIA FOR INITIAL APPROVAL
A. Bile acid synthesis disorders due to single enzyme defects (SEDs)
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:
1. The diagnosis is confirmed by mass spectrometry or other biochemical testing, genetic
testing, or enzyme assay.
2. The member has liver dysfunction (i.e., elevated transaminases, elevated bilirubin,
presence of cholestasis) at baseline.
B. Peroxisomal disorders (PDs) including Zellweger spectrum disorders
Authorization of 6 months may be granted for adjunctive treatment of peroxisomal disorders
when both of the following criteria are met:
1. The diagnosis is confirmed by mass spectrometry or other biochemical testing, or by
genetic testing.
2. The member exhibits manifestations of liver disease (i.e., elevated transaminases,
elevated bilirubin, presence of cholestasis).
IV. CONTINUATION OF THERAPY
Authorization of 12 months may be granted to members with an indication listed in Section III
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:
- Cholbam [package insert]. San Diego, CA: Manchester Pharmaceuticals, Inc. a wholly owned subsidiary of Travere Therapeutics, Inc.; March 2023.
- 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. 2009;137:1310-1320.
- Heubi J, Setchell KDR, Bove KE. Inborn errors of bile acid metabolism. Seminars Liver Dis. 2007;27:282-294.
- 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.
April 17, 2024
