CurrentmhnPolicy CP.PHAR.283

Clinical Policy: Lomitapide (Juxtapid)

Pharmacy clinical policy for coverage and prior authorization of lomitapide (Juxtapid) for homozygous familial hypercholesterolemia (HoFH) across Commercial and Medicaid lines of business; includes initial and continuation criteria, dosing limits, contraindications, REMS, and related appendices.

Policy Summary

Payermhn

PolicyClinical Policy: Lomitapide (Juxtapid)

Policy CodePolicy CP.PHAR.283

Change TypeAnnual reviews (2021-2025) — criteria revisions and additions

Effective Date10.01.16

Next Review Date

Key ActionProvider must submit documentation supporting all approval criteria including recent LDL-C within 60 days, genetic or clinical evidence of HoFH, adherence to statin/ezetimibe, and specialist involvement.

POLICY UPDATE CHANGES

1Q 2025 annual review lowered untreated LDL requirement to 400 mg/dL and revised evidence of HeFH in both parents to evidence of familial hypercholesterolemia in at least one parent; references reviewed and updated.

1Q 2024 annual review added Leqvio to list of drugs where coadministration is not allowed and required that 'Treatment plan does not include coadministration with Leqvio, Repatha, or Praluent' also for continuation.

1Q 2023 annual review lowered minimum LDL requirement to 55 mg/dL for members with ASCVD at very high risk and added Appendix H.

1Q 2021 annual review added requirement for adherence to statin therapy on re-auth.

1Q 2022 annual review removed references to withdrawn products and updated capsule availability per PI.

1FDA approved indication (HoFH)

60 mg/dayMaximum daily dose

6 monthsInitial approval duration

REMSREMS requirement present

Coverage Summary

Policy stance: Juxtapid (lomitapide) is covered with prior authorization under defined criteria for treatment of homozygous familial hypercholesterolemia (HoFH). Coverage requires documentation that the member meets the HoFH-specific clinical and laboratory criteria, specialist prescribing/consultation, adherence to lipid‑lowering therapies and other listed requirements; initial approval duration is 6 months. The medication is restricted to its FDA‑approved HoFH indication; non‑FDA (off‑label) uses are handled per the plan's off‑label policies and are not authorized by this policy except when supported through those off‑label review processes.

Key thresholds

Treated LDL-C threshold for HoFH≥ 300 mg/dL

Treated non-HDL-C threshold for HoFH≥ 330 mg/dL

Untreated LDL-C threshold for HoFH≥ 400 mg/dL

On-treatment LDL-C requirement (recent within 60 days)≥ 70 mg/dL

On-treatment LDL-C for very high ASCVD risk (recent within 60 days)≥ 55 mg/dL

Maximum daily dose60 mg per day / 2 capsules per day

Initial Therapy Criteria - Homozygous Familial Hypercholesterolemia

Initial Approval Criteria - Homozygous Familial Hypercholesterolemia

Covered when ALL of the following are met:

ALL of the following

ONE of
- Genetic mutation indicating HoFH (e.g., mutations in LDLR, PCSK9, apoB, LDLRAP1)
- Treated LDL-C ≥ 300 mg/dL or non-HDL-C ≥ 330 mg/dL≥ 300 mg/dL (LDL-C); ≥ 330 mg/dL (non-HDL-C)
- ALL of the following
  - ONE of

Initial Approval Criteria - Other diagnoses/indications

If not meeting HoFH criteria:

If drug has undergone a recent label change within last 6 months not reflected in this policy, follow formulary/no-coverage or non-formulary policy references or, if not applicable, refer to off-label use policies

Refer to CP.CPA.190, CP.PMN.255, CP.PMN.16 for formulary/no-coverage or non-formulary routing; CP.CPA.09 (commercial) or CP.PMN.53 (Medicaid) for off-label uses.

Continuation (Reauthorization) Criteria - Homozygous Familial Hypercholesterolemia

Continuation (Reauthorization) Criteria - Homozygous Familial Hypercholesterolemia (must meet all):

{

"operator":"all",

"children":[

{"text":"a. Currently receiving medication via Centene benefit or member has previously met initial approval criteria."},

b. Member is currently receiving medication and is enrolled in a state and product with continuity of care regulations (refer to state specific addendums for CC.PHARM.03A and CC.PHARM.03B).

Continuation Criteria - Other diagnoses/indications

Same routing as initial 'Other diagnoses' section

Refer to formulary/no-coverage or non-formulary policies if recent label change; otherwise refer to off-label use policy

Refer to CP.CPA.190, CP.PMN.255, CP.PMN.16; or CP.CPA.09 (commercial) / CP.PMN.53 (Medicaid).

Not Authorized / Unapproved Uses

Not Authorized

Non-FDA approved indications not addressed in this policy are not authorized unless sufficient documentation per off-label use policies or evidence of coverage documents is provided

Refer to CP.CPA.09 (commercial) or CP.PMN.53 (Medicaid).

Safety and REMS implications: Juxtapid is available only through a REMS program because of a boxed hepatotoxicity risk; the REMS requirement and associated hepatotoxicity monitoring/documentation must be followed as part of authorization and ongoing management.

Applicable Codes

Product Codes / Dosage FormsNDC|mixed

Capsules 5 mg	Lomitapide 5 mg capsule
Capsules 10 mg	Lomitapide 10 mg capsule
Capsules 20 mg	Lomitapide 20 mg capsule
Capsules 30 mg	Lomitapide 30 mg capsule

Provider Actions & Requirements

Prior Authorization

Documentation required for approval

Submit documentation supporting all approval criteria: office chart notes, laboratory results with an LDL-C measured within the last 60 days, genetic testing or clinical evidence of HoFH (per criteria), documentation of adherence to statin and ezetimibe where applicable, and evidence of prescribing by or consultation with a cardiologist, endocrinologist, or lipid specialist.

Office chart notes demonstrating diagnosis and clinical course
Recent LDL-C result within 60 days
Genetic test results or clinical evidence of HoFH (e.g., mutations in LDLR, PCSK9, apoB, LDLRAP1 or documented criteria such as untreated LDL-C ≥ 400 mg/dL with supporting family/xanthoma history)
Documentation of adherence to statin and ezetimibe regimens (when applicable)
Documentation of specialist prescribing or consultation

Clinical Evidence & References

Evidence references: Prescribing information cited as: Juxtapid Prescribing Information; September 2020 (product PI). Guideline influence: the 2022 ACC expert consensus decision pathway informed LDL thresholds and is noted as influencing policy updates (e.g., changes reflected in 1Q2023 and 1Q2025 reviews).

Background & Definitions

Background: Lomitapide is a microsomal triglyceride transfer protein (MTP) inhibitor indicated as an adjunct to a low‑fat diet and other lipid‑lowering treatments to reduce LDL‑C and related lipids in patients with homozygous familial hypercholesterolemia (HoFH). The safety and effectiveness of Juxtapid have not been established in patients without HoFH (including HeFH). Because of a boxed hepatotoxicity warning, Juxtapid is available only through the Juxtapid REMS Program. Dosing is initiated at 5 mg PO QD with titration per the PI and a maximum daily dose of 60 mg/day; product capsules are available in 5 mg, 10 mg, 20 mg, and 30 mg strengths.

Term	Definition
{"text":"HoFH","status":""},{"text":"Homozygous familial hypercholesterolemia","status":""}
{"text":"HeFH / FH","status":""},{"text":"Heterozygous familial hypercholesterolemia / familial hypercholesterolemia","status":""}
{"text":"SAMS","status":""},{"text":"Statin-associated muscle symptoms","status":""}
{"text":"REMS","status":""},{"text":"Risk Evaluation and Mitigation Strategy (Juxtapid REMS Program due to hepatotoxicity risk)","status":""}

Revision History

1Q 2025policy changeLatest

Per 2022 ACC expert consensus decision pathway, lowered untreated LDL requirement to 400 mg/dL and revised evidence of HeFH in both parents to evidence of familial hypercholesterolemia in at least one parent; references reviewed and updated. (P&T Approval Date = 02.25)

1Q 2024policy change

Added Leqvio to list of drugs where coadministration is not allowed and required that 'Treatment plan does not include coadministration with Leqvio, Repatha, or Praluent' also for continuation; Appendix I clarified smoking is specific to tobacco and revised HeFH to FH; references reviewed and updated. (P&T Approval Date = 02.24)

1Q 2023

Policy Summary

Payermhn

PolicyClinical Policy: Lomitapide (Juxtapid)

Policy CodePolicy CP.PHAR.283

Change TypeAnnual reviews (2021-2025) — criteria revisions and additions

Effective Date10.01.16

Next Review Date

On This Page

Tendinous or cutaneous xanthoma prior to age 10 years

Evidence of familial hypercholesterolemia in at least one parent (e.g., documented history of elevated LDL-C ≥ 190 mg/dL prior to lipid-lowering therapy)LDL-C ≥ 190 mg/dL in parent

Prescribed by or in consultation with a cardiologist, endocrinologist, or lipid specialist

See provider actions for documentation requirement.

Age ≥ 18 years≥ 18 years

ANY of the following

LDL-C ≥ 70 mg/dL≥ 70 mg/dL
LDL-C ≥ 55 mg/dL if member has ASCVD and is at very high risk (see Appendix H)≥ 55 mg/dL

For members on statin therapy, both of the following must be met

Juxtapid is prescribed in conjunction with a statin at the maximally tolerated dose
Appendix D defines high/moderate/low intensity regimens.
ALL of the following
- A high intensity statin (see Appendix D)
- ANY of the following
  - Intolerance to two high intensity statins
  - A statin risk factor (see Appendix F)
- ANY of the following
  - Intolerance to one high and one moderate intensity statins
  - A statin risk factor (see Appendix F) and history of intolerance to two moderate intensity statins

For members who are statin intolerant, both of the following

Statin therapy is contraindicated per Appendix E
Member has tried at least two statins, one of which must be hydrophilic (pravastatin, fluvastatin, or rosuvastatin)
ALL of the following
- Documentation of re-challenge with titration from lowest possible dose and/or intermittent dosing frequency (e.g., 1 to 3 times weekly)

Member has been adherent to ezetimibe therapy used concomitantly with a statin at the maximally tolerated dose for at least the last 4 months, unless contraindicated per Appendix E or member has a history of ezetimibe intolerance≥ 4 months adherence

Failure of a preferred PCSK9 inhibitor, if applicable, unless contraindicated or clinically significant adverse effects are experienced

Treatment plan does not include coadministration with Leqvio, Repatha, or Praluent

Coadministration prohibition also required for continuation.

Dose does not exceed both: 60 mg per day and 2 capsules per day≤ 60 mg/day; ≤ 2 capsules/day

Maximum dose per dosing/availability.

],"operator":"any"},

If request is for a dose increase, new dose does not exceed: a. 60 mg per day; b. 2 capsules per day.

Documentation Required

Approval duration and monitoring

Initial authorization: 6 months. For continuation, documentation of LDL-C response is required within the last 3 months showing a reduction from pre‑treatment baseline.

Initial authorization duration: 6 months.
For reauthorization, submit LDL-C lab results obtained within the last 3 months that demonstrate a reduction in LDL-C from the baseline value prior to initiation of Juxtapid.
If reauthorization is requested for a dose increase, document new dose does not exceed 60 mg/day and 2 capsules/day.
Document adherence to concomitant statin (if statin tolerant) and ezetimibe per policy; include clinic notes or pharmacy fill history as applicable.

Prior Authorization

Specialist prescribing/consultation requirement

For initial approval, Juxtapid must be prescribed by or in consultation with a cardiologist, endocrinologist, or lipid specialist. Documentation of the specialist prescriber or consult should be included with the request.

Documentation Required

Adherence evidence

Provide documentation of at least 4 months of adherence to a maximally tolerated statin regimen. If ezetimibe is used concomitantly with a statin, document at least 4 months of adherence to that combination unless contraindicated or intolerant.

At least 4 months adherence to maximally tolerated statin dose prior to approval
At least 4 months adherence to concomitant ezetimibe with a statin when applicable, unless contraindicated or intolerant

Prior Authorization

PCSK9 inhibitor failure requirement

If applicable, the member must have failed a preferred PCSK9 inhibitor prior to approval unless the PCSK9 is contraindicated or the member experienced clinically significant adverse effects. Document failure or contraindication/toxicities to the preferred PCSK9 agent.

Documented failure of a preferred PCSK9 inhibitor, or
Documented contraindication or clinically significant adverse effects to PCSK9 inhibitors

Documentation Required

REMS and hepatotoxicity risk

Juxtapid is available only through the Juxtapid REMS Program because of hepatotoxicity risk. Providers must follow REMS enrollment and monitoring/documentation requirements specified for the product when requesting coverage.

Enrollment in the Juxtapid REMS Program is required
Follow REMS-specific monitoring and documentation for hepatotoxicity

Denial Risk

Non-FDA use not authorized

Requests for non‑FDA approved indications not addressed in this policy will be denied unless sufficient documentation per the off‑label use policies (CP.CPA.09 for commercial or CP.PMN.53 for Medicaid) or evidence of coverage documents is provided.

Per 2022 ACC expert consensus decision pathway, lowered minimum LDL requirement to 55 mg/dL for members with ASCVD at very high risk and added corresponding Appendix H; references reviewed and updated. (P&T Approval Date = 02.23)

1Q 2022policy change

Removed references to withdrawn products (Kynamro) and removed 40 mg and 60 mg capsules per updated PI; references reviewed and updated. (P&T Approval Date = 02.22)

1Q 2021policy change

Added requirement for adherence to statin therapy on re-authorization; references reviewed and updated. (Date = 11.02.20; P&T Approval Date = 02.21)