CurrentAvMedPolicy N/A

Cholbam

Pharmacy prior authorization form and clinical criteria for coverage of Cholbam (cholic acid) for bile acid synthesis disorders due to single enzyme defects and select peroxisomal disorders for AvMed members; includes initial and reauthorization requirements, dosing guidance, quantity limits, monitoring, and specialty pharmacy note.

Policy Summary

PayerAvMed

PolicyCholbam

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionPrescribing physician must sign and print name on request and fax completed form with required documentation (labs, diagnostics, chart notes) to 1-305-671-0200.

SourceLink

POLICY UPDATE CHANGES

No material clinical/coverage changes reported.

6Initial authorization duration (months)

12Reauthorization duration (months)

>=3 weeksMinimum age for eligibility

2Primary covered condition groups

Coverage Summary

Scope: Pharmacy prior authorization form and clinical criteria for coverage of Cholbam (cholic acid) for bile acid synthesis disorders due to single enzyme defects (SEDs) and select peroxisomal disorders for AvMed members. Coverage stance: covered_with_criteria. Dosing guidance: 10–15 mg/kg once daily or in 2 divided doses (use 11–17 mg/kg once daily or in 2 divided doses for patients with concomitant familial hypertriglyceridemia). Specialty pharmacy dispensing: medication is provided by PropriumRx and requires specialty pharmacy handling.

Reauthorization thresholds and durations

Age>= 3 weeks

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

Total bilirubin (reauth)<= 1 mg/dL

Body weight response (reauth)Increase by 10% or remains > 50th percentile

Initial authorization duration2 months

Reauthorization duration12 months

Reauthorization / Continued Therapy Criteria

Reauthorization numeric thresholds (badges)

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

Total bilirubin reauth thresholdTotal bilirubin <= 1 mg/dL

Body weight reauth thresholdBody weight increased by 10% or remains > 50th percentile

Provider Actions & Authorization Requirements

Prior Authorization

Submit pharmacy prior authorization form

The prescribing physician must sign and clearly print their name (preprinted stamps not valid) on the completed pharmacy prior authorization form. Fax the completed form with required documentation (labs, diagnostics, chart notes) to 1-305-671-0200. Incomplete, incorrect, or illegible information may delay the authorization process.

Documentation Required

Provide diagnostic confirmation

Provide diagnostic confirmation by submitting mass spectrometry (FAB-MS) serum or urinary bile acid levels and/or molecular genetic testing results for the PEX gene as applicable to support the diagnosis.

Documentation Required

Liver function monitoring

Assessment of liver function (AST, ALT, and bilirubin) must be performed initially and will be performed with each renewal; submit lab results with the request.

Billing Rule

Specialty pharmacy dispensing

Medication must be dispensed by the specialty pharmacy PropriumRx.

Denial Risk

Concomitant therapy with BSEP inhibitors

Approval requires the member not be on concomitant therapy with Bile Salt Efflux Pump (BSEP) inhibitors (e.g., cyclosporine). If concomitant therapy is unavoidable, the member must be monitored closely for adverse reactions; failure to document monitoring or unsafe/unmonitored concomitant use may result in denial.

Step Therapy

Use of samples

Use of samples to initiate therapy does not meet step edit or preauthorization criteria and therefore does not satisfy step therapy/preauthorization requirements.

Background

Cholbam (cholic acid) is indicated as replacement therapy for bile acid synthesis disorders due to single enzyme defects and as adjunctive treatment in certain peroxisomal disorders. For single enzyme defects, diagnoses supported include 3‑beta‑hydoxysteroid dehydrogenase (3‑β‑HSD) deficiency, AKR1D1 deficiency, cerebrotendinous xanthomatosis (CTX), and AMACR deficiency; diagnosis must be confirmed by mass spectrometry (FAB‑MS) of serum or urinary bile acid levels. For peroxisomal disorders, diagnoses include neonatal adrenoleukodystrophy, generalized peroxisomal disorder, Refsum disease, Zellweger syndrome, or peroxisomal disorder type unknown; confirmation may be by FAB‑MS or pathogenic PEX gene variants, and the medication is used as adjunctive treatment when the member exhibits at least one manifestation such as liver disease, steatorrhea, or complications from decreased fat‑soluble vitamin absorption.

Recommended dosing: Oral 10 to 15 mg/kg once daily or in two divided doses; administer 11 to 17 mg/kg once daily or in two divided doses in patients with concomitant familial hypertriglyceridemia. Quantity limits per available strengths are noted (e.g., 50 mg – 4 capsules/day; 250 mg – 7 capsules/day).

Monitoring and safety: Members must be ≥ 3 weeks of age for initiation; assessment of liver function (AST, ALT, and bilirubin) must be performed initially and with each renewal and submitted with requests. Members must not be receiving treatment for extrahepatic (neurologic) manifestations to meet criteria for coverage. Concomitant therapy with Bile Salt Efflux Pump (BSEP) inhibitors (e.g., cyclosporine) is not allowed unless unavoidable; if used, close monitoring for adverse reactions is required and lack of monitoring may result in denial.

Specialty pharmacy: Medication is dispensed by specialty pharmacy PropriumRx. Use of samples to initiate therapy does not meet preauthorization criteria.

FAB‑MS: Fast atom bombardment mass spectrometry used to analyze serum or urinary bile acid levels for diagnosis of bile acid synthesis disorders.

BSEP inhibitors: Drugs that inhibit the bile salt export pump (for example, cyclosporine) which may interact with Cholbam therapy and require monitoring if concomitant use is unavoidable.

Policy Summary

PayerAvMed

PolicyCholbam

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionPrescribing physician must sign and print name on request and fax completed form with required documentation (labs, diagnostics, chart notes) to 1-305-671-0200.

SourceLink

On This Page

50 mg - 4 capsules per day	Maximum daily capsules for 50 mg strength
250 mg - 7 capsules per day	Maximum daily capsules for 250 mg strength

OpenPayer

Cholbam

Coverage Summary

Initial Therapy — Single Enzyme Defects (SEDs)

Initial Therapy — Peroxisomal Disorders

Reauthorization / Continued Therapy Criteria

Provider Actions & Authorization Requirements

Applicable Codes & Quantity Limits

Background

Clinical Evidence

Revision History