CurrentmhnPolicy CP.PHAR.390

Cholic acid (Cholbam)

Defines medical necessity criteria, initial and continuation approval, dosing limits, documentation requirements, and approval durations for Cholbam (cholic acid) for bile acid synthesis disorders due to single enzyme defects and peroxisomal disorders including Zellweger spectrum disorders across Commercial, HIM, and Medicaid lines of business.

Policy Summary

Payermhn

PolicyCholic acid (Cholbam)

Policy CodePolicy CP.PHAR.390

Change TypeAnnual review; administrative revisions

Effective DateDec 1, 2018

Next Review Date

Key ActionProvider must submit documentation (office chart notes, lab results, or other clinical information) supporting that member meets all approval criteria.

SourceLink

POLICY UPDATE CHANGES

4Q 2025 annual review: no significant changes; references reviewed and updated.

4Q 2024 annual review: no significant changes; removed supplemental information in Appendix D; references reviewed and updated.

Revised continued therapy approval duration for Commercial line of business from length of benefit to 12 months or duration of request, whichever is less.

4Q 2021 annual review: no significant changes; references to HIM.PHAR.21 revised to HIM.PA.154; references reviewed and updated.

2Covered Indications (FDA)

17 mg/kg/dayMaximum Dose

12 monthsApproval Duration (Medicaid/HIM)

12 monthsApproval Duration (Commercial)

Coverage Summary

Defines medical necessity criteria, initial and continued approval, dosing limits, documentation requirements, and approval durations for Cholbam (cholic acid) for bile acid synthesis disorders due to single enzyme defects (SEDs) and peroxisomal disorders (PDs) including Zellweger spectrum disorders. The policy requires confirmed diagnosis (e.g., abnormal urinary bile acids by FAB‑MS or molecular genetic testing), specialist prescribing or consultation, current liver function tests for initiation, and a dose limit of ≤ 17 mg/kg per day. Approval durations are 12 months for Medicaid/HIM and 12 months or duration of request for Commercial lines of business.

Key thresholds and approval durations

Maximum dose17 mg/kg per day

ALT/AST response (continued therapy)ALT or AST < 50 U/L OR >= 80% reduction from baseline

Total bilirubin response (continued therapy)Total bilirubin <= 1 mg/dL

LFT recency for initial approvalDocumentation of current liver function tests within the last 30 days

Approval Duration (Medicaid/HIM)12 months

Approval Duration (Commercial)12 months or duration of request, whichever is less

Initial Therapy Criteria

I. Initial Approval Criteria - Bile Acid Synthesis Disorders or Peroxisomal Disorders

Covered when ALL of the following are met:

ALL of the following

ONE of
- Bile acid synthesis disorders due to single enzyme defects (SEDs)
- Peroxisomal disorders (PDs), including Zellweger spectrum disorders
ONE of
- An abnormal urinary bile acid consistent with a bile acid synthesis or Zellweger spectrum disorder as confirmed by fast atom bombardment ionization - mass spectrometry (FAB-MS) analysis

Continuation Therapy Criteria

II. Continued Therapy - Bile Acid Synthesis Disorders or Peroxisomal Disorders

Continued therapy is approved when ALL of the following are met:

ALL of the following

ONE of
- Currently receiving medication via Centene benefit or member has previously met initial approval criteria
- Member is currently receiving medication and is enrolled in a state and product with continuity of care regulations (refer to state specific addendums CC.PHARM.03A and CC.PHARM.03B)
ALL of the following
- Alanine transaminase (ALT) or aspartate transaminase (AST) values reduced to < 50 U/L or baseline levels reduced by 80%

Diagnoses / Indications Not Authorized

III. Diagnoses/Indications Not Authorized

Non-FDA approved indications that are not addressed in this policy are not authorized unless there is sufficient documentation of efficacy and safety according to off-label use policies

Refer to CP.CPA.09 (commercial), HIM.PA.154 (marketplace), CP.PMN.53 (Medicaid)

The policy notes that the safety and effectiveness of Cholbam for extrahepatic manifestations of bile acid synthesis disorders or peroxisomal disorders have not been established. Requests for uses not covered by this policy (non‑FDA approved or not specifically listed) will be managed according to the applicable off‑label use policies for the relevant line of business.

Provider Actions & Prior Authorization Requirements

Prior Authorization

Specialist prescribing requirement

Cholbam initial prescriptions must be written by or in consultation with a hepatologist, gastroenterologist, or metabolic disease specialist.

Documentation Required

Submit supporting documentation

Submit office chart notes, laboratory results, or other clinical information supporting that the member meets all approval criteria. Initial approval requires current liver function test results within the last 30 days.

Applicable Codes & Product Availability

Product availability (NDC not listed)mixed

Capsule: 50 mg	Cholbam capsule strength available
Capsule: 250 mg	Cholbam capsule strength available

Clinical Evidence & References

Evidence cited includes the Cholbam Prescribing Information (Retrophin, October 2024). The policy and references were reviewed through the 4Q 2025 annual review with no significant changes noted. No additional trial‑specific quantitative metrics are provided in the brief.

Background and Definitions

Cholic acid (Cholbam) is FDA‑approved for treatment of bile acid synthesis disorders due to single enzyme defects and as adjunctive therapy for peroxisomal disorders, including Zellweger spectrum disorders, when patients have manifestations of liver disease, steatorrhea, or fat‑soluble vitamin malabsorption. The product is available as 50 mg and 250 mg capsules. The safety and effectiveness for extrahepatic manifestations have not been established.

Term	Definition
FAB-MS	fast atom bombardment ionization - mass spectrometry
SEDs	single enzyme defects
PDs	peroxisomal disorders

Revision History

11.25.25annual reviewLatest

4Q 2025 annual review: no significant changes; references reviewed and updated.

11.24annual review

4Q 2024 annual review: no significant changes; removed supplemental information in Appendix D; references reviewed and updated.

02.22policy revision (material)

Revised continued therapy approval duration for Commercial line of business from length of benefit to 12 months or duration of request, whichever is less (change dated 10.18.21; P&T Approval Date = 02.22).

Policy Summary

Payermhn

PolicyCholic acid (Cholbam)

Policy CodePolicy CP.PHAR.390

Change TypeAnnual review; administrative revisions

Effective DateDec 1, 2018

Next Review Date

Key ActionProvider must submit documentation (office chart notes, lab results, or other clinical information) supporting that member meets all approval criteria.

SourceLink

On This Page

Molecular genetic testing consistent with diagnosis (e.g., biallelic pathogenic variants in ABCD3, AKR1D1, AMACR, HSD3B7, CYP27A1, CYP7B, or PEX genes)

Prescribed by or in consultation with a hepatologist, gastroenterologist, or metabolic disease specialist

Documentation of current (within the last 30 days) liver function test resultswithin the last 30 days

Dose does not exceed 17 mg/kg per day<= 17 mg/kg per day

I. Initial Approval Criteria - Other diagnoses/indications

Covered when ONE of the following applies:

ANY of the following

If the drug had a label change within the last 6 months not reflected in policy, follow the relevant formulary/no-coverage or non-formulary policies for the line of business
Refer to CP.CPA.190, HIM.PA.33, CP.PMN.255 for formulary/no-coverage or CP.CPA.190, HIM.PA.103, CP.PMN.16 for non-formulary as applicable
If requested use is NOT listed under section III and criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business
Refer to CP.CPA.09 (commercial), HIM.PA.154 (marketplace), CP.PMN.53 (Medicaid)

ALT or AST < 50 U/L OR >= 80% reduction from baseline

Total bilirubin values reduced to ≤ 1 mg/dLTotal bilirubin <= 1 mg/dL

If request is for a dose increase, new dose does not exceed 17 mg/kg per day<= 17 mg/kg per day

II. Continued Therapy - Other diagnoses/indications

Continued therapy for other indications follows the same referral logic as initial other diagnoses/indications:

ANY of the following

If the drug had a label change within the last 6 months not reflected in policy, follow relevant formulary/no-coverage or non-formulary policies (references provided)
Refer to CP.CPA.190, HIM.PA.33, CP.PMN.255 for formulary/no-coverage or CP.CPA.190, HIM.PA.103, CP.PMN.16 for non-formulary as applicable
If requested use is NOT listed under section III and criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business
Refer to CP.CPA.09 for commercial, HIM.PA.154 for marketplace, and CP.PMN.53 for Medicaid

Documentation Required

Liver function tests required

Initial approval requires documentation of current liver function tests within the last 30 days. Continued therapy requires evidence of improvement in liver function tests: ALT or AST reduced to < 50 U/L or reduced by 80% from baseline, and total bilirubin reduced to ≤ 1 mg/dL.

Billing Rule

Dose limit enforcement

Dose must not exceed 17 mg/kg per day for initial therapy or for any dose increase. For patients with concomitant familial hypertriglyceridemia, dosing up to 11–17 mg/kg/day is specified (maximum 17 mg/kg/day).

Denial Risk

Non-FDA or non-specified uses

Requests for indications not listed in this policy (non‑FDA or other non‑specified uses) are not authorized and will be denied unless sufficient documentation of efficacy and safety is provided in accordance with the applicable off‑label use policy for the line of business.

Documentation Required

Discontinuation criteria

Discontinue Cholbam if liver function does not improve within 3 months of starting treatment or if complete biliary obstruction develops.

11.21annual review

4Q 2021 annual review: no significant changes; references to HIM.PHAR.21 revised to HIM.PA.154; references reviewed and updated (date = 06.28.21; P&T Approval Date = 11.21).