CurrentUnitedHealthcarePolicy N/A

Cholbam (cholic acid) prior authorization

Prior authorization criteria and reauthorization requirements for Cholbam (cholic acid) for members covered under UnitedHealthcare Pharmacy Clinical Pharmacy Programs.

Policy Summary

PayerUnitedHealthcare

PolicyCholbam (cholic acid) prior authorization

Policy CodePolicy N/A

Change TypeNo material change

Effective DateAug 1, 2025

Next Review DateN/A

Key ActionSubmit initial prior authorization showing diagnosis of a bile acid synthesis disorder due to single enzyme defects OR diagnosis of a peroxisomal disorder with liver manifestations and use as adjunctive therapy.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

8/1/2025policy effective date

SEDs & PDsFDA-indicated populations

3 monthsdiscontinue if no improvement

12 monthsauthorization duration

Coverage Criteria

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Initial Authorization

Pathway A: Diagnosis of a bile acid synthesis disorder due to single enzyme defects (SEDs)
Pathway B: Diagnosis of a peroxisomal disorder including Zellweger spectrum disorders; patient exhibits manifestations of liver disease, steatorrhea, or complications from decreased fat‑soluble vitamin absorption; Cholbam is being used as adjunctive treatment

Treatment of extrahepatic manifestations of bile acid synthesis disorders or peroxisomal disorders is not an established indication for Cholbam. The policy follows the product limitation stating that the safety and effectiveness for extrahepatic manifestations have not been established, and coverage is limited to the FDA‑indicated hepatic-related populations and adjunctive use described elsewhere in this policy.

Initial Therapy

Initial approval criteria

Initial therapy

Pathway A: Both of the following: (1) Diagnosis of a bile acid synthesis disorder; (2) Bile acid synthesis disorder is due to single enzyme defects (SEDs)
Pathway B: All of the following: (1) Diagnosis of a peroxisomal disorder including Zellweger spectrum disorders; (2) Patient exhibits manifestations of liver disease, steatorrhea, or complications from decreased fat‑soluble vitamin absorption; (3) Cholbam is being used as adjunctive treatment

Reauthorization / Continuation Criteria

Reauthorization/Continuation

Conditions required for reauthorization/continuation

Continuation therapy: Continuation authorized when positive clinical response documented and no complete biliary obstruction12 months

Authorization will be issued for 12 months

Provider Actions and Requirements

Prior Authorization

Initial Authorization Required

Providers must obtain initial prior authorization and submit required documentation to support the requested therapy.

Required documentation: diagnosis, indication (SED or peroxisomal disorder with liver disease/steatorrhea), treatment intent (adjunctive when applicable).

Note

Automated approval / claim-based authorization possible

UnitedHealthcare may use automated approval or claim-based authorization pathways — approvals can be granted based on prior claim/medication history, ICD-10 diagnosis codes, and claim logic without additional documentation when program rules allow.

Automated approvals vary by clinical program and payer implementation.
Supply limits may apply and will be enforced through claims processing.

Documentation Required

Reauthorization — documentation of positive clinical response required

For reauthorization, submit documentation demonstrating a positive clinical response to Cholbam therapy.

Evidence should include improvement in liver function tests (e.g., AST, ALT) and clinical status.
Confirm absence of complete biliary obstruction at reauthorization.
Document time-on-therapy and any other relevant labs or clinical notes supporting benefit.

Denial Risk

Clinical signs that may trigger discontinuation or denial

Monitor and discontinue therapy when clinical signs indicate lack of benefit or safety concerns to avoid denial or termination of coverage.

Discontinue if there is no improvement in liver function within 3 months of treatment initiation.
Discontinue if complete biliary obstruction develops.
Discontinue or consider denial if there are persistent clinical or laboratory indicators of worsening liver function or cholestasis (for example, rising AST/ALT, worsening bilirubin, clinical deterioration).

Definitions

Extrahepatic manifestations — limitation of use note

DefinitionExtrahepatic manifestations: clinical features outside the liver associated with bile acid synthesis disorders or peroxisomal disorders (including Zellweger spectrum disorders).

Limitation of useThe safety and effectiveness of Cholbam on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects (SEDs) or peroxisomal disorders (PDs) including Zellweger spectrum disorders have not been established.

Implication for coverageCholbam is indicated for hepatic manifestations of SEDs and as adjunctive therapy for hepatic manifestations of PDs; use for extrahepatic manifestations is not supported by established safety/effectiveness data.

Treatment discontinuation considerationsTherapy should be discontinued if liver function does not improve within 3 months, if complete biliary obstruction develops, or with persistent/worsening liver dysfunction or cholestasis.

Background

Cholbam (cholic acid) is a bile acid replacement therapy indicated for treatment of bile acid synthesis disorders due to single enzyme defects (SEDs) and as adjunctive therapy for certain peroxisomal disorders (including Zellweger spectrum disorders) in patients with manifestations of liver disease, steatorrhea, or complications from decreased fat‑soluble vitamin absorption. Clinical response should be monitored with liver function testing, and therapy should be discontinued if there is no improvement in liver function within 3 months, if complete biliary obstruction develops, or with persistent or worsening liver dysfunction or cholestasis.