ModifiedmhnPolicy CP.PHAR.287

Obeticholic Acid (Ocaliva) for Primary Biliary Cholangitis

Defines medical necessity, continued therapy, contraindications, and administration details for obeticholic acid (Ocaliva) for members treated for primary biliary cholangitis (PBC) across commercial, HIM, and Medicaid lines of business.

Policy Summary

Payermhn

PolicyObeticholic Acid (Ocaliva) for Primary Biliary Cholangitis

Policy CodePolicy CP.PHAR.287

Change TypeRemoved initial approvaladded continued-therapy attestation

Effective DateNov 16, 2023

Next Review Date

Key ActionFor continued therapy, submit prior authorization with provider attestation acknowledging the manufacturer's voluntary withdrawal and documentation of ALP response.

SourceLink

POLICY UPDATE CHANGES

Initial approval criteria for new starts of Ocaliva were removed due to the manufacturer's voluntary withdrawal from the US market.

Provider attestation acknowledging the manufacturer's voluntary withdrawal is required for continued therapy.

UDCA was clarified to be ursodiol in appendices/notes.

12 monthsapproval duration for continued therapy

10 mg/daymaximum daily dose

5 mg, 10 mgtablet strengths

Withdrawal

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Coverage Criteria

Continued Therapy (Primary Biliary Cholangitis)"

Covered when ALL of the following are met for continued therapy in PBC patients:

Continued therapy requirements: 1) Provider attestation acknowledging the manufacturer's voluntary withdrawal of Ocaliva from the US market; 2) Member meets ONE of the following: (a) currently receiving medication via Centene benefit or has previously met initial approval criteria, OR (b) member is currently receiving medication and is enrolled in a state/product with continuity of care regulations; 3) Member is responding positively to therapy as evidenced by ONE of the following: (a) initial reauthorization — reduction in ALP level from pretreatment level, OR (b) subsequent reauthorization — continued reduction or maintenance of the initial reduction in ALP level; 4) If request is for a dose increase, the new dose must not exceed BOTH: (a) 10 mg per day, AND (b) 1 tablet per day.See node text (ALP response criteria referenced in Appendix D)

Approval duration: 12 months

Initial Therapy (Primary Biliary Cholangitis)"

Initial therapy:

Initial approval: Authorization is not permitted; member may not initiate therapy with Ocaliva due to the manufacturer's voluntary withdrawal from the US market.N/A

Approval duration: Not applicable

Other Indications / Off-label Routing"}]}009

Other diagnoses/indications:

Alternate pathways: 1) If the drug has undergone a recent label change (within the last 6 months) not yet reflected in this policy, follow the specified formulary or non‑formulary policies for the relevant line of business (see CP.CPA.190, HIM.PA.33, CP.PMN.255 for formulary; CP.CPA.190, HIM.PA.103, CP.PMN.16 for non‑formulary). 2) If the requested use is not listed under exclusions and criterion 1 does not apply, refer to the off‑label use policy for the relevant line of business (CP.CPA.09 for commercial, HIM.PA.154 for HIM, CP.PMN.53 for Medicaid).N/A

Refer to the listed policies per line of business

Non–FDA approved indications that are not specifically addressed in this policy are not authorized unless the provider supplies sufficient documentation of safety and efficacy in accordance with the applicable off‑label use policy for the member's line of business (see CP.CPA.09 for Commercial, HIM.PA.154 for Health Insurance Marketplace, and CP.PMN.53 for Medicaid) or per the member's evidence of coverage documents.

Obeticholic acid (Ocaliva) is contraindicated in patients with decompensated cirrhosis (e.g., Child‑Pugh Class B or C) or a history of a prior decompensation event. It is also contraindicated in patients with compensated cirrhosis who have evidence of portal hypertension (for example ascites, gastroesophageal varices, or persistent thrombocytopenia), and in those with complete biliary obstruction. The policy includes a boxed warning that hepatic decompensation and liver failure, including fatal outcomes or need for liver transplant, have been reported with Ocaliva in PBC patients with compensated or decompensated cirrhosis; permanently discontinue Ocaliva for laboratory or clinical evidence of hepatic decompensation, new evidence of portal hypertension in compensated cirrhosis, or clinically significant hepatic adverse reactions.

Coverage determinations are governed by the member's applicable coverage documents (for example, evidence of coverage, certificate of coverage, policy, or contract of insurance) and by state and federal requirements. Providers must follow those terms, conditions, exclusions, and limitations when requesting authorization; adherence to plan provisions and applicable policies is required for benefits administration and may affect coverage decisions.

Because the manufacturer voluntarily withdrew Ocaliva from the U.S. market, initiation of Ocaliva is not permitted and requests to start therapy will be considered not medically necessary. This market withdrawal also underpins requirements limiting any authorizations to continuation of therapy for members already receiving the drug.

Coding and Measurement Thresholds

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

No codes listed

ALP trial thresholds — response criteria

Trial response — composite endpointALP < 1.67 x ULN AND total bilirubin ≤ ULN AND ALP decrease ≥ 15% from baseline (trial-defined composite response)

ALP decrease thresholdAt least 15% reduction in ALP from pretreatment level

ALP absolute thresholdALP less than 1.67 × ULN

ULN values used in trialULN for ALP defined as 118 U/L for females and 124 U/L for males

Maximum daily dose

Maximum daily dose (policy)10 mg/day

Dosing regimen context

Provider Actions and Authorization Requirements

Prior Authorization

Prior Authorization for Continued Therapy

Prior authorization is required for continuation of therapy only — initial authorizations for new starts are not permitted because the manufacturer voluntarily withdrew Ocaliva from the U.S. market. Providers should not request initiation of Ocaliva; requests will be denied for new starts.

Initial authorization for new therapy: Not permitted (manufacturer withdrawal)
Continued therapy: Prior authorization required for members currently receiving Ocaliva

Note

Prior Authorization Requirement and Coverage Administration

Providers must follow the Health Plan's medical necessity guidelines and submit requests through the plan's prior authorization process. Coverage decisions and administration of benefits are subject to the terms, conditions, exclusions, and limitations of the member's coverage documents and to applicable state and federal requirements.

Submit requests per applicable prior authorization workflows and forms
Coverage is governed by evidence of coverage/certificate of coverage and Health Plan administrative policies

Background

Obeticholic acid (Ocaliva) is a farnesoid X receptor agonist that received an accelerated approval pathway for adult patients with primary biliary cholangitis (PBC), intended either as add‑on therapy to ursodeoxycholic acid (UDCA/ursodiol) for inadequate responders or as monotherapy for those intolerant to UDCA. Continued approval was based on reductions in alkaline phosphatase (ALP); improvement in survival or clinical symptoms has not been established. Following regulatory concerns about liver safety, the manufacturer voluntarily withdrew Ocaliva for PBC from the U.S. market, which led to removal of initial‑start authorizations and constrained use to select continuing patients under this policy.

Definitions

ALP — definition and ULN values used in trials

DefinitionALP (alkaline phosphatase): a liver enzyme used to assess response in PBC

ULN values (trial)ULN for ALP defined as 118 U/L for females and 124 U/L for males

Role in monitoringUsed in composite trial endpoint and for assessing treatment response (absolute and percent change)

UDCA — clarified as ursodiol

Acronym expandedUDCA = ursodeoxycholic acid

Common name (clarification)UDCA clarified as ursodiol

Recommended dosing (AASLD)

Revision History

2025-09-17policy_revisionLatest

Added disclaimer about manufacturer withdrawal, removed initial approval criteria, and added requirement for provider attestation for continued therapy.

2025-08-25P&T_approval

P&T approval: added disclaimer about manufacturer withdrawal with removal of initial approval criteria and addition of provider attestation requirement for continued therapy.

2025-06-27