Subject: Cholbam 2119-A SGM P2023
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
transminases, 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 the diagnosis is
confirmed by mass spectrometry or other biochemical testing
or genetic testing, and the member exhibits manifestations
of liver disease.
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 are experiencing benefit from therapy as
evidenced by improvement from baseline as documented per
clinical chart notes.
A. Bile acid synthesis disorders due to single enzyme
defects (SEDs)
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization for bile
acid synthesis disorders due to single enzyme defects who
have achieved and maintained improvement in liver function
(i.e., reduced transaminases, reduced bilirubin, no evidence
of cholestasis on liver biopsy).
B. Peroxisomal disorders (PDs) including Zellweger
spectrum disorders
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization for
adjunctive treatment of peroxisomal disorders with Cholbam
who have achieved and maintained improvement in liver
function (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: Retrophin, Inc.; October 2020.
- 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.
June 18, 2023
