CurrentCentenePolicy CP.PHAR.390

Cholic Acid (Cholbam) Therapy

Defines medical necessity criteria, documentation, dosing limits, and coverage determinations for cholic acid (Cholbam) for members across Centene lines of business (Commercial, HIM, Medicaid). Applies to prescribers and reviewers making coverage decisions.

Policy Summary

PayerCentene

PolicyCholic Acid (Cholbam) Therapy

Policy CodePolicy CP.PHAR.390

Change TypeNo material changes

Effective DateDec 1, 2018

Next Review Date

Key ActionSubmit prior authorization with supporting documentation (office notes, labs, diagnostic confirmation, and prescriber specialty) showing member meets all approval criteria.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

17maximum allowed dose

12approval duration (months)

FAB-MS/geneticdiagnostic confirmation

Specialistprescriber specialty

2018publication year

Centene

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Coverage Criteria for Cholic Acid (Cholbam)

Initial Therapy

Covered when ALL of the following are met

Initial Approval - Bile Acid Synthesis Disorders or Peroxisomal Disorders: 1. Diagnosis of one of the following: a) bile acid synthesis disorders due to single enzyme defects (SEDs); OR b) peroxisomal disorders (PDs), including Zellweger spectrum disorders; 2. Diagnosis confirmed by at least one of the following: a) an abnormal urinary bile acid profile consistent with a bile acid synthesis or Zellweger spectrum disorder by fast atom bombardment ionization - mass spectrometry (FAB-MS); OR b) molecular genetic testing consistent with the diagnosis (e.g., biallelic pathogenic variants in ABCD3, AKR1D1, AMACR, HSD3B7, CYP27A1, CYP7B, or PEX genes); 3. Prescribed by or in consultation with a hepatologist, gastroenterologist, or metabolic disease specialist; 4. Documentation of current (within the last 30 days) liver function test results; 5. Dose does not exceed 17 mg/kg per day.dose <= 17 mg/kg/day

Use FAB-MS or molecular genetic testing for diagnostic confirmation

Continuation Therapy

Covered when ALL of the following are met

Continued Therapy - Bile Acid Synthesis Disorders or Peroxisomal Disorders: 1. Member meets one of the following: a) currently receiving medication via Centene benefit or previously met initial approval criteria; OR b) currently receiving medication and enrolled in a state/product with continuity-of-care regulations; 2. Member is responding positively to therapy as evidenced by BOTH: a) ALT or AST values reduced to < 50 U/L or baseline reduced by 80%; AND b) total bilirubin reduced to ≤ 1 mg/dL; 3. If request is for a dose increase, the new dose does not exceed 17 mg/kg per day.LFT and bilirubin response thresholds

Discontinue if liver function does not improve within 3 months of starting treatment

Non-FDA approved indications that are not specifically addressed in this policy are not authorized unless the request includes sufficient documentation of efficacy and safety in accordance with the applicable off‑label use policies: CP.CPA.09 (Commercial), HIM.PA.154 (Health Insurance Marketplace), and CP.PMN.53 (Medicaid).

When state Medicaid coverage provisions conflict with the coverage provisions in this clinical policy, state Medicaid coverage provisions take precedence. Refer to the applicable state Medicaid manual for any state‑specific coverage requirements.

The safety and effectiveness of cholic acid (Cholbam) for extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects (SEDs) or peroxisomal disorders (PDs) have not been established. Requests for non‑FDA indications lacking sufficient evidence or off‑label documentation per the referenced off‑label policies are considered not authorized.

Coding and Laboratory Thresholds

No explicit CPT/HCPCS/ICD-10/NDC codes listed in this sectionmixed

No codes listed

LFT response criteria — ALT/AST and total bilirubin

ALT/AST responseALT or AST values reduced to < 50 U/L or baseline reduced by 80%

Total bilirubin responseTotal bilirubin values reduced to ≤ 1 mg/dL

Timing for LFTsDocumentation of current (within the last 30 days) liver function test results required

Maximum daily dose

Maximum daily doseDose must not exceed 17 mg/kg/day

Typical dosing range10–15 mg/kg/day administered orally in one or two divided doses for SEDs and PDs

Provider Actions, Authorization & Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for initial and continued therapy. Requests must meet the policy's clinical criteria for approval. Approval durations: Medicaid/HIM — 12 months; Commercial — 12 months or duration of request, whichever is less.

Initial and continued therapy clinical criteria must be met
Approval durations: Medicaid/HIM — 12 months; Commercial — 12 months or duration of request, whichever is less

Documentation Required

Required Documentation

Providers must submit documentation (e.g., office chart notes, lab results, or other clinical information) supporting that the member meets all approval criteria when requesting authorization.

Include documentation of diagnostic confirmation (e.g., abnormal urinary bile acids by FAB-MS or molecular genetic testing)

Background

Cholic acid (Cholbam) is a naturally occurring bile acid indicated for the treatment of bile acid synthesis disorders due to single enzyme defects (SEDs) and as adjunctive therapy for peroxisomal disorders (PDs), including Zellweger spectrum disorders, when hepatic manifestations or malabsorption are present. Diagnosis may be confirmed by an abnormal urinary bile acid profile assessed by fast atom bombardment mass spectrometry (FAB‑MS) or by molecular genetic testing identifying pathogenic variants consistent with the disorder. Per Appendix D, discontinue Cholbam if liver function does not improve within 3 months of treatment or if complete biliary obstruction develops.

Definitions and Abbreviations

FAB-MS

Termfast atom bombardment ionization - mass spectrometry (FAB-MS)

Use in diagnosisFAB-MS is used to confirm an abnormal urinary bile acid profile consistent with bile acid synthesis or Zellweger spectrum disorders

RoleAssesses the phenotypic, biochemical response to a genetic disorder

Definitions — SEDs and PDs

SEDsSEDs = single enzyme defects (bile acid synthesis disorders)

PDsPDs = peroxisomal disorders, including Zellweger spectrum disorders

Indication context

Policy Summary

PayerCentene

PolicyCholic Acid (Cholbam) Therapy

Policy CodePolicy CP.PHAR.390

Change TypeNo material changes

Effective DateDec 1, 2018

Next Review Date

Key ActionSubmit prior authorization with supporting documentation (office notes, labs, diagnostic confirmation, and prescriber specialty) showing member meets all approval criteria.

SourceLink

On This Page

Concomitant familial hypertriglyceridemia dosing11–17 mg/kg/day PO in one or two divided doses (maximum still 17 mg/kg/day)

Include specialty prescriber or consult (hepatologist, gastroenterologist, or metabolic disease specialist)

Include current (within 30 days) liver function test results

Denial Risk

Documentation-based Denial Risk

Requests that lack required documentation may be denied. Examples of documentation-based denial risks include failure to provide confirmatory diagnostic testing, absence of specialty prescriber/consult information, or missing recent liver function tests demonstrating response.

Missing confirmatory diagnostic evidence (FAB‑MS urinary bile acid results or molecular genetic testing)
No documentation of specialty prescriber or consultation
No recent (within 30 days) liver function test results or lack of evidence of biochemical response for continuation requests

Note

Medicaid Precedence

For Medicaid members, state Medicaid coverage provisions supersede this clinical policy where they conflict. Providers should refer to the applicable state Medicaid manual for specific coverage rules.

State Medicaid provisions take precedence over this policy when in conflict
Refer to state-specific Medicaid manuals and addendums

Note

Contractual Obligations & Submission Requirements

This clinical policy is the property of the Health Plan. Providers, members, and their representatives are bound by the terms and conditions herein through contract or by submitting claims for payment.

Providers must follow Health Plan clinical policy requirements when submitting claims
Unauthorized copying or distribution of this policy is prohibited

Cholic acid indicated for treatment of SEDs and as adjunctive treatment for PDs with hepatic or malabsorption manifestations

Placeholder term(No term provided in source document)

Policy ownershipThis clinical policy is the property of the Health Plan; terms and conditions govern use and distribution

Contractual bindingProviders, members, and representatives are bound by policy terms when submitting claims or providing services