Lomitapide (Juxtapid) for homozygous familial hypercholesterolemia — Coverage Criteria
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Defines medical necessity, prior authorization requirements, and documentation for Juxtapid (lomitapide) used as adjunct therapy in patients with homozygous familial hypercholesterolemia (HoFH). Applies to Wellmark/Blue Cross Blue Shield members covered under applicable contracts.
No material clinical or coverage changes in this revision.
Coverage Criteria for Juxtapid (lomitapide)
Initial Approval (primary pathway)
Covered when ALL of the following are met (primary pathway):
See Appendix A/B for diagnostic criteria
If high-intensity statins are not tolerated, two moderate-intensity statin trials with ezetimibe are acceptable
REMS enrollment required
Initial Approval (statin-intolerance/contraindication pathway)
Alternative covered pathway when statin intolerance or contraindication is present:
See Appendix A/B for diagnostic criteria
Statin intolerance requires inability to tolerate at least two statins (one at any dose, one at the lowest daily dose) with reproducible symptoms/biomarkers and appropriate re-challenge as described in the policy; each statin trial (including re-challenge) should be at least two weeks.
Continuation of Therapy
Covered when ALL of the following are met for continuation:
Documentation of untreated baseline LDL and current LDL (within 90 days) is required.
Definite FH diagnostic criteria
Definite diagnosis of familial hypercholesterolemia is made when ONE of the following is met:
Genetic testing confirms pathogenic variant
Clinical thresholds and signs per Simon-Broome diagnostic criteria
Use Dutch scoring system for diagnosis
The safety and effectiveness of Juxtapid (lomitapide) have not been established for use in patients with hypercholesterolemia who do not have homozygous familial hypercholesterolemia (HoFH), including individuals with heterozygous familial hypercholesterolemia (HeFH).
When identifying HoFH, clinicians should exclude secondary causes of hypercholesterolemia; the clinical rationale emphasizes that HoFH is characterized by severely elevated LDL-C in the absence of secondary contributors to high cholesterol.
Juxtapid is considered not medically necessary for patients who do not meet the specific coverage criteria outlined above. Documentation requirements (including LDL-C measurements) are required to support medical necessity determinations.
No specific CPT, HCPCS, revenue, or ICD-CM codes are provided in this policy section for Juxtapid administration or related services; the procedures and billing codes field is listed as Not applicable.
Procedures, Codes, and Clinical Thresholds
| No codes listed |
Authorization, Documentation, and REMS Requirements
Prior Authorization Required
Prior authorization required. Approval will be for 12 months; quantity limit of 30 capsules per 30 days may apply. Continuation requires meeting initial criteria and demonstration of LDL‑C reduction ≥ 20% from pre‑treatment levels.
- Prior authorization approval period: 12 months
- Quantity limit: Juxtapid 30 capsules per 30 days
REMS and Hepatotoxicity Risk
Use of Juxtapid carries a risk of hepatotoxicity and is available only through an FDA Risk Evaluation and Mitigation Strategy (REMS). Prescribers must be enrolled in and comply with REMS requirements prior to initiation and for the duration of therapy. Documentation of provider REMS enrollment and adherence to required monitoring (including baseline and ongoing liver function testing) must be submitted with the authorization request.
- REMS enrollment required for prescriber
- Monitor and document baseline and ongoing hepatic tests per REMS guidance
Diagnostic Documentation Required
Documentation must include evidence supporting a diagnosis of homozygous familial hypercholesterolemia (HoFH) and prior/ongoing lipid‑lowering therapy. Acceptable documentation for diagnosis includes genetic testing demonstrating mutations in both alleles of LDLR, ApoB, PCSK9, or LDLRAP1 OR clinical diagnostic criteria (Simon‑Broome or Dutch Lipid Clinic Network criteria) or the Appendix criteria (e.g., untreated LDL‑C > 400 mg/dL with early xanthomas or parental definite FH).
- Genetic testing report OR clinical criteria (Simon‑Broome or Dutch Lipid Clinic Network)
- Appendix evidence (e.g., untreated LDL‑C > 400 mg/dL, xanthomas before age 10, parental definite FH)
Required Laboratory and Therapy Documentation
Provide untreated baseline LDL, LDL while on statin therapy (prior to initiating Juxtapid), and current LDL (within 90 days). Include chart notes demonstrating statin intolerance or contraindication when applicable, and medical records supporting prior trials of lipid‑lowering therapy (including PCSK9‑targeted therapy).
- Untreated baseline LDL value
- LDL on statin therapy prior to Juxtapid initiation
- Current LDL within 90 days of request
- Chart notes and records showing statin intolerance or contraindication (if applicable)
- Documentation of prior PCSK9‑targeted therapy trial
Adjunctive Therapy Requirements
Juxtapid is authorized only as adjunctive therapy to maximally tolerated lipid‑lowering regimens and LDL apheresis where applicable, not as first‑line monotherapy. Initial approval requires documented trials of required lipid‑lowering therapies: either high‑intensity statin (atorvastatin 40–80 mg or rosuvastatin 20–40 mg) plus ezetimibe (or two moderate‑intensity statin trials with ezetimibe if high‑intensity statins are not tolerated) AND a trial of a PCSK9‑targeted therapy unless contraindicated. Members must continue concomitant lipid‑lowering therapy while receiving Juxtapid; Juxtapid is not to be used in combination with PCSK9‑targeted therapy.
- Adjunct to maximally tolerated lipid‑lowering therapy and LDL apheresis as applicable
- Required trials: high‑intensity statin + ezetimibe (or two moderate‑intensity statin trials + ezetimibe if high‑intensity not tolerated)
- Trial of PCSK9‑targeted therapy required unless contraindicated
- Do not combine Juxtapid with PCSK9‑targeted therapy
- Continue concomitant lipid‑lowering therapy during Juxtapid treatment
Definitions and Diagnostic Criteria
Background and Clinical Context
Lomitapide (Juxtapid) is indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce LDL-C, total cholesterol, apolipoprotein B, and non-HDL-C in patients with homozygous familial hypercholesterolemia (HoFH). The effect on cardiovascular morbidity and mortality is undetermined, and safety and effectiveness have not been established in patients without HoFH.
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