CurrentCentenePolicy CP.PHAR.633

Eplontersen (Wainua) (PDF)

Defines medical necessity criteria, dosing, approval durations, exclusions, and coding implications for eplontersen (Wainua) for treatment of hereditary transthyretin-mediated (TTR) amyloidosis with polyneuropathy across Commercial, HIM, and Medicaid lines of business.

Policy Summary

PayerCentene

PolicyEplontersen (Wainua) (PDF)

Policy CodePolicy CP.PHAR.633

Change TypeMaterial updates (criteria and coding)

Effective Date12.21.23

Next Review Date

Key ActionProvider must submit documentation (office notes, lab results, etc.) supporting that member meets all approval criteria.

SourceLink

POLICY UPDATE CHANGES

HCPCS codes C9399 and J3490 were added to the policy.

Removed criteria 'member has not received prior treatment with Onpattro or Amvuttra' per competitor analysis.

Criteria updated per FDA labeling when drug became FDA approved.

Annual reviews updated references with no significant clinical changes.

1FDA approved indication(s)

45 mg/monthMaximum dose

≥18Minimum age

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Coverage Summary & Scope

Coverage stance: Covered with criteria. Subject: Eplontersen (Wainua). Scope: defines medical necessity criteria, dosing, approval durations, exclusions, and coding implications for treatment of hereditary transthyretin-mediated (TTR) amyloidosis with polyneuropathy across Commercial, HIM, and Medicaid. Status: CURRENT. Effective date: 2023-12-21. Last review date: 2025-05-25. Key thresholds: Maximum monthly dose: 45 mg once monthly; Age: ≥ 18 years.

Initial Therapy Criteria

I. Initial Approval Criteria - Hereditary Transthyretin-Mediated Amyloidosis

Covered when ALL of the following are met:

Diagnosis of hereditary TTR-mediated amyloidosis with polyneuropathy

Documentation confirms presence of a TTR mutation

DNA sequencing is usually required for genetic confirmation (see appendices)

Biopsy is positive for amyloid deposits OR medical justification is provided as to why treatment should be initiated despite a negative biopsy or no biopsy

Demonstration of amyloid deposits via tissue biopsy (eg, Congo red staining) is essential; negative biopsy does not exclude disease

Prescribed by or in consultation with a neurologist

Age ≥ 18 years≥ 18 years

Member has not had a prior liver transplant

Wainua is not prescribed concurrently with Onpattro or Amvuttra

Concurrent therapy exclusion may result in denial

Dose does not exceed 45 mg once monthlyMaximum monthly dose: 45 mg

Dosing regimen for hereditary TTR-mediated amyloidosis with polyneuropathy is 45 mg SC once monthly

I. Initial Approval Criteria - Other diagnoses/indications

Covered under limited circumstances (must meet ONE of the following):

If this drug has recently (within the last 6 months) undergone a label change (eg, newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, follow formulary/no-coverage policy (CP.CPA.190 for commercial, HIM.PA.33 for marketplace, CP.PMN.255 for Medicaid) or non-formulary policy (CP.CPA.190 for commercial, HIM.PA.103 for marketplace, CP.PMN.16 for Medicaid) as appropriate

Refer to CP.CPA.190, HIM.PA.33, CP.PMN.255, HIM.PA.103, CP.PMN.16 for operational guidance

If the requested use (eg, diagnosis, age, dosing regimen) is NOT specifically listed under section III 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 marketplace, CP.PMN.53 for Medicaid)

Refer to CP.CPA.09, HIM.PA.154, CP.PMN.53 for off-label operational guidance

Continuation (Renewal) Criteria

II. Continuation (Renewal) Criteria - Hereditary TTR-mediated amyloidosis

Covered for continuation when ALL of the following are met:

Member currently receiving medication via Centene benefit or previously met initial approval criteria OR member is currently receiving medication and enrolled in a state/product with continuity of care regulations

Refer to state specific addendums CC.PHARM.03A and CC.PHARM.03B

Member is responding positively to therapy as evidenced by improvement in any of: measures of polyneuropathy (motor strength, sensation, reflexes), quality of life, motor function, walking ability, or nutritional status (eg, modified mass index)

Evidence of clinical benefit required for renewal

Member has not had a prior liver transplant

Wainua is not prescribed concurrently with Onpattro or Amvuttra

Concurrent therapy exclusion may result in denial

Diagnoses/Indications Not Authorized

III. Diagnoses/Indications NOT authorized

Non-FDA approved indications not addressed in this policy are not authorized unless sufficient documentation of efficacy and safety according to off-label use policies (CP.CPA.09 for commercial, HIM.PA.154 for marketplace, CP.PMN.53 for Medicaid) or evidence of coverage documents is provided

Concurrent therapy exclusion: Wainua must not be prescribed concurrently with Onpattro or Amvuttra; claims may be denied if prescribed concurrently (see provider actions / denial risk).

Applicable Codes

HCPCS codes referencedHCPCS

C9399	Unclassified drugs or biologicals
J3490	Unclassified drugs

Provider Actions & Billing Requirements

Prior Authorization

Prior authorization required

Requests are subject to the stated prior authorization criteria; approval durations vary by line of business (see Approval duration).

Documentation Required

Submit supporting documentation

Provider must submit documentation (such as office chart notes, lab results or other clinical information) supporting that member has met all approval criteria.

Documentation Required

Biopsy or justification required

Provide positive biopsy for amyloid deposits or medical justification for treatment despite negative/no biopsy.

Clinical Evidence & References

Key evidence includes the NEURO-TTRansform trial reported in JAMA (2023) demonstrating efficacy for hereditary TTR amyloidosis with polyneuropathy, and the Wainua Prescriber Information (AstraZeneca; September 2024).

Background & Definitions

Background: Eplontersen (Wainua) is a transthyretin-directed antisense oligonucleotide indicated for treatment of polyneuropathy of hereditary TTR-mediated amyloidosis in adults. Typical diagnostic components include genetic confirmation via DNA sequencing and tissue biopsy demonstrating amyloid deposits.

Definitions: TTR = transthyretin; FDA = Food and Drug Administration.

Revision History

05.01.23creation

Policy created pre-emptively (policy created pre-emptively, Date = 05.01.23).

01.09.24FDA approval update

Drug became FDA approved; criteria updated per FDA labeling and HCPCS codes C9399 and J3490 were added to the policy.

02.12.24administrative update

2Q 2024 annual review: no significant clinical changes; references reviewed and updated.