Currentsierra health and lifePolicy 2025D0104I

Evkeeza (evinacumab-dgnb) for homozygous familial hypercholesterolemia (HoFH)

Medical benefit drug policy specifying coverage criteria for Evkeeza (evinacumab-dgnb) as adjunct therapy for treatment of HoFH (adults and pediatric patients ≥5 years) including initial and continuation authorization requirements, applicable codes, dosing conformity, and restrictions (e.g., not with lomitapide).

Policy Summary

Payersierra health and life

PolicyEvkeeza (evinacumab-dgnb) for homozygous familial hypercholesterolemia (HoFH)

Policy CodePolicy 2025D0104I

Change TypeApplicable diagnosis code updates

Effective DateOct 1, 2025

Next Review Date

Key ActionObtain prior authorization demonstrating diagnosis of HoFH by/with a lipid specialist, confirmation via genetic testing OR specified untreated LDL-C and additional history, document prior therapies/intolerances, and plan to continue background LDL‑C lowering therapies; initial authorization limited to ≤ 12 months.

POLICY UPDATE CHANGES

Added ICD-10 codes E78.010, E78.011, and E78.019 to the applicable diagnosis list.

1Drug covered indication (HoFH)

≤12 monthsAuthorization duration (initial and renewal)

5+FDA indicated age (>= 5 years)

Coverage Summary

Treatment must follow FDA-approved dosing and Evkeeza must not be used in combination with Juxtapid (lomitapide). Applicable billing/code references include HCPCS J1305 and ICD-10 codes added to the diagnosis list (e.g., E78.010, E78.011, E78.019).

Key thresholds and quick facts

FDA indicated ageApproved as adjunct therapy for HoFH in adults and pediatric patients aged 5 years and older

Untreated LDL-C threshold for clinical confirmationUntreated LDL-C greater than 400 mg/dL (used as part of HoFH diagnostic criteria)

Treatment LDL-C goal referenced for failureFailure to achieve an LDL-C goal of < 55 mg/dL despite specified background therapy is a criterion for Evkeeza

Authorization durationInitial and reauthorization approvals are each limited to no more than 12 months

Initial Therapy Criteria

Initial Therapy

Evkeeza is proven and medically necessary for HoFH patients who meet ALL of the following criteria:

Diagnosis confirmation
- Genetic confirmation: Submission of medical records confirming genetic confirmation of biallelic pathogenic/likely pathogenic variants on different chromosomes at the LDLR, APOB, PCSK9, or LDLRAP1 genes or ≥ 2 such variants at different loci
- Clinical features (both required)
  - Supporting clinical feature: One of the following: Xanthoma before 10 years of age, Evidence of familial hypercholesterolemia in at least one parent
Age or LDL-C response
- Age under 10: Patient is less than 10 years of age
- Failure to achieve LDL-C goal
  - Statin status: One of the following: Patient is currently treated with maximally tolerated statin therapy; OR Patient is unable to tolerate statin therapy as evidenced by intolerable and persistent (> 2 weeks) symptoms (Myalgia without CK elevations; Myositis with CK elevations < 10× ULN; Rhabdomyolysis or muscle symptoms with CK elevations > 10× ULN; OR contraindication to all statins)
    Maximally tolerated statin therapy definition per Definitions section
Treatment administration and combination constraints
- Background therapy: Patient will continue other traditional LDL‑C lowering therapies (e.g., maximally tolerated statins, ezetimibe) in combination with Evkeeza
- No lomitapide combination: Evkeeza will not be used in combination with Juxtapid (lomitapide)
- Dosing per FDA: Evkeeza dosing is in accordance with the United States Food and Drug Administration approved labeling
- Initial authorization duration: Initial authorization will be for no more than 12 months≤ 12 months

Root: Top-level AND

Continuation Therapy (Reauthorization)

Continuation of Therapy (Reauthorization)

Reauthorization requires ALL of the following:

Reauthorization criteria

Positive clinical response: Documentation of a positive clinical response to Evkeeza therapy (see Definitions)
Positive clinical response defined in Definitions section
No lomitapide combination: Evkeeza will not be used in combination with Juxtapid (lomitapide)
Dosing per FDA: Evkeeza dosing is in accordance with the United States Food and Drug Administration approved labeling
Reauthorization duration: Reauthorization will be for no more than 12 months≤ 12 months

Root: Top-level AND

Additional Considerations / Alternate Pathways

Additional Considerations / Alternate Pathways (Background clinical qualifiers)

Additional (sometimes Medicare-specific) acceptable alternatives or qualifiers include any of the following:

Additional qualifiers

Ezetimibe: Patient has been receiving ezetimibe therapy as adjunct to maximally tolerated statin therapy; or patient has a history of intolerance or contraindication to ezetimibe
PCSK9 pathway: One of the following: Patient has been treated with PCSK9 therapy or did not respond to PCSK9 therapy; OR physician attests patient is known to have two LDL-receptor negative alleles and therefore would not respond to PCSK9 therapy; OR patient has a history of intolerance or contraindication to PCSK9 therapy
Prior lomitapide: Patient has previously been treated with Juxtapid (lomitapide)
Prior apheresis: Patient has previously been treated with lipoprotein apheresis

Root: Top-level any

Maximally tolerated statin therapy: Use of statin at the highest dose tolerated by the patient; for Medicare reviews refer to CMS section as applicable.

Positive clinical response: Documentation demonstrating clinical benefit from Evkeeza therapy (as judged by the prescribing clinician), required for reauthorization.

Applicable Codes

Drug HCPCSHCPCSCovered

J1305

Injection, evinacumab-dgnb, 5 mg

Applicable ICD-10 diagnosis codes (HoFH / FH related)ICD-10

E78.010	Homozygous familial hypercholesterolemia [HoFH]
E78.011	Heterozygous familial hypercholesterolemia [HeFH]
E78.019	Familial hypercholesterolemia, unspecified
Z83.42	Family history of FH

Provider Actions & Prior Authorization

Prior Authorization

Initial authorization requirements

Obtain prior authorization documenting HoFH diagnosis by or with a lipid specialist, confirmation by genetic testing or specified clinical criteria (untreated LDL‑C > 400 mg/dL with xanthoma before age 10 or parental FH), documentation of prior therapies/intolerances, and plan to continue background LDL‑C lowering therapies. Initial approvals are limited to no more than 12 months.

Affected codes: J1305, E78.010, E78.011, E78.019, Z83.42
Initial authorization ≤ 12 months

Prior Authorization

Reauthorization requirements

For reauthorization, provide documentation of a positive clinical response to Evkeeza, confirm dosing per FDA labeling, and verify Evkeeza is not being used with lomitapide. Reauthorizations are limited to no more than 12 months.

Affected code: J1305
Reauthorization ≤ 12 months

Documentation Required

Genetic and clinical confirmation documentation

Submit medical records (chart notes, laboratory values) to support genetic confirmation of biallelic pathogenic/likely pathogenic variants or, alternatively, untreated LDL‑C > 400 mg/dL plus supporting history such as xanthoma before age 10 or evidence of familial hypercholesterolemia in a parent.

Required records: chart notes, laboratory values
Acceptable evidence: genetic confirmation of biallelic pathogenic/likely pathogenic variants OR untreated LDL‑C > 400 mg/dL plus xanthoma before age 10 or parental familial hypercholesterolemia

Billing Rule

Dosing and combination constraints

Ensure Evkeeza dosing conforms to FDA‑approved labeling and do not co‑administer with lomitapide (Juxtapid).

Affected code: J1305
Do not combine Evkeeza with Juxtapid (lomitapide)

Clinical Evidence

-48%Mean percent change in LDL-C at week 24 in pediatric study (ages 5–11); 95% CI: -69% to -28%

Evinacumab-dgnb is a recombinant human monoclonal antibody that binds and inhibits ANGPTL3 (an anti-ANGPTL3 mAb), a regulator of lipoprotein metabolism that affects lipoprotein lipase- and endothelial lipase-mediated hydrolysis of triglycerides and phospholipids.

Evkeeza is indicated as an adjunct to other LDL-C lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with HoFH.

Clinical trials demonstrated substantial LDL-C reductions: the phase 3 ELIPSE HoFH trial showed a between-group least-squares mean percent difference in LDL-C at week 24 of -49.0 percentage points (absolute difference -132.1 mg/dL, p < 0.001), and the pediatric study (ages 5–11) reported a mean percent change in LDL-C at week 24 of -48% (95% CI: -69% to -28%).

Background

Homozygous familial hypercholesterolemia (HoFH) is characterized by markedly elevated LDL-C and premature cardiovascular risk due to bi-allelic pathogenic mutations (loss-of-function variants in the LDL receptor pathway) leading to low or absent LDL clearance; if untreated, mortality is common before age 30.

Diagnosis can be supported by genetic confirmation of biallelic pathogenic/likely pathogenic variants in LDLR, APOB, PCSK9, or LDLRAP1 (or ≥ 2 such variants at different loci), or by clinical features such as untreated LDL-C > 400 mg/dL, xanthoma before age 10, or evidence of familial hypercholesterolemia in at least one parent.

Evkeeza (evinacumab-dgnb) is approved as an adjunct therapy for HoFH (patients aged 5 years and older) and acts by binding and blocking ANGPTL3 activity to lower triglycerides and LDL-C processing upstream of LDL formation.

Revision History

2025-10-01code_addedLatest