ModifiedmhnPolicy CP.PHAR.287

Clinical Policy: Obeticholic Acid (Ocaliva)

Defines medical necessity, initial and continued therapy criteria, contraindications, dosing, and administrative requirements for obeticholic acid (Ocaliva) across Commercial, HIM, and Medicaid lines of business. Also documents manufacturer voluntary withdrawal from the US market and resulting restrictions on new starts.

Policy Summary

Payermhn

PolicyClinical Policy: Obeticholic Acid (Ocaliva)

Policy CodePolicy CP.PHAR.287

Change TypeManufacturer voluntary withdrawal added; continued-therapy restrictions

Effective DateNov 16, 2011

Next Review Date

Key ActionProvider must submit office notes, lab results, or other clinical information supporting that member has met all approval criteria.

SourceLink

POLICY UPDATE CHANGES

Added step therapy bypass for IL HIM per IL HB 5395 and clarified UDCA is ursodiol; references reviewed and updated.

Added disclaimer about manufacturer withdrawal with removal of initial approval criteria and added requirement for provider attestation to continued therapy.

Updated indication to 'without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension' and removed Child Pugh B/C dosing as contraindicated per PI.

1Covered Indication (PBC, limited continued therapy only)

0New starts allowed

10 mg/dayMaximum Dose

Coverage Summary

Ocaliva (obeticholic acid) is covered with criteria only for continued therapy in primary biliary cholangitis (PBC) because the manufacturer voluntarily withdrew Ocaliva for PBC from the US market; new starts are not permitted. The policy effective date is 11.16 and the last review date is 8.25. The scope of this policy applies to Commercial, HIM, and Medicaid lines of business.

Initial Therapy Criteria

I. Initial Approval Criteria

Provider must submit documentation supporting that member has met all approval criteria. (All criteria must be met.)

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Provider Actions & Administrative Requirements

Documentation Required

Submit supporting documentation

Submit office notes, lab results, or other clinical information supporting that the member has met all approval criteria.

Prior Authorization

No new starts authorized

Authorization for initiation of Ocaliva is not permitted due to the manufacturer's voluntary withdrawal; providers must not request initiation for new patients.

Documentation Required

Attestation required for continued therapy

Provider must attest that they acknowledge the manufacturer's voluntary withdrawal of Ocaliva from the US market for members requesting continued therapy.

Documentation Required

Demonstrate biochemical response

For reauthorization, document a reduction in alkaline phosphatase (ALP) from pretreatment level for initial reauthorization, and continued reduction or maintenance of that initial ALP reduction for subsequent reauthorization.

Billing Rule

Dose increase limits

Dose increase requests will be denied if the new dose exceeds 10 mg per day or more than 1 tablet per day.

Step Therapy

Refer to formulary/non-formulary/off-label policies

Follow the referenced formulary, non-formulary, or off-label policies for prior authorization pathways per line of business.

CP.CPA.190
HIM.PA.33
CP.PMN.255
HIM.PA.103
CP.PMN.16
CP.CPA.09
HIM.PA.154
CP.PMN.53

Clinical Evidence

compositeTrial composite response: ALP <1.67x ULN AND total bilirubin ≤ ULN AND ALP decrease ≥15%

ALP F/MALP ULN used in trial: females 118 U/L; males 124 U/L

Tbili F/MTotal bilirubin ULN used in trial: females 1.1 mg/dL; males 1.5 mg/dL

Ocaliva received accelerated approval based on reduction in alkaline phosphatase (ALP), and confirmatory trials were required to verify clinical benefit. The clinical trial defined response as a composite of: ALP < 1.67 x ULN AND total bilirubin ≤ ULN AND ALP decrease ≥ 15%. The ULN values used in the trial were specified as: ALP ULN: females 118 U/L, males 124 U/L; total bilirubin ULN: females 1.1 mg/dL, males 1.5 mg/dL.

Background & Definitions

Ocaliva is a farnesoid X receptor (FXR) agonist approved for adult patients with primary biliary cholangitis (PBC) either in combination with ursodeoxycholic acid (UDCA) for adults with an inadequate response to UDCA, or as monotherapy for adults unable to tolerate UDCA. The indication was approved under the accelerated approval pathway based on ALP reduction, with confirmatory trials required. Intercept Pharmaceuticals announced the voluntary withdrawal of Ocaliva for PBC from the US market in September 2025, which affects the Commercial, HIM, and Medicaid lines of business covered by this policy.

Term	Definition
ALP
Alkaline phosphatase
PBC
Primary biliary cholangitis
UDCA
Ursodeoxycholic acid (ursodiol)

Revision History

04.13.22 / P&T 08.22annual_review

3Q 2022 annual review: updated indication to 'without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension' and removed Child Pugh B/C dosing as contraindicated per PI.

06.27.25 / P&T 08.25annual_review

3Q 2025 annual review: added step therapy bypass for IL HIM per IL HB 5395 and clarified that UDCA refers to ursodiol; references reviewed and updated.

09.17.25policy_changeLatest

Change reflecting voluntary withdrawal of Ocaliva from the US market (manufacturer announcement): added disclaimer about withdrawal, removed initial approval criteria prohibiting new starts, and added requirement for provider attestation to continued therapy. Marked material.

Policy Summary

Payermhn

PolicyClinical Policy: Obeticholic Acid (Ocaliva)

Policy CodePolicy CP.PHAR.287

Change TypeManufacturer voluntary withdrawal added; continued-therapy restrictions

Effective DateNov 16, 2011

Next Review Date

Key ActionProvider must submit office notes, lab results, or other clinical information supporting that member has met all approval criteria.

SourceLink

On This Page

OpenPayer

Clinical Policy: Obeticholic Acid (Ocaliva)

Coverage Summary

Initial Therapy Criteria

Continuation Therapy Criteria

Unapproved Indications / Exclusions

Provider Actions & Administrative Requirements

Applicable Codes & Identifiers

Clinical Evidence

Background & Definitions

Revision History