CurrentCignaPolicy IP0107

Zokinvy

Cigna coverage policy for prescription benefit coverage of Zokinvy (lonafarnib capsules) specifying prior authorization, FDA-approved indication criteria for Hutchinson-Gilford Progeria Syndrome (HGPS), exclusions for other progeroid conditions, documentation and prescriber requirements, and genetic mutation appendix.

Policy Summary

PayerCigna

PolicyZokinvy

Policy CodePolicy IP0107

Change TypeAnnual revisions / documentation clarifications

Effective Date

Next Review Date

Key ActionPrior Authorization is required for Zokinvy; provide required documentation (age, BSA ≥ 0.39 m2, LMNA genetic testing confirming a pathogenic variant, and specialist consultation) for a 1-year approval.

POLICY UPDATE CHANGES

Documentation language added: 'Documentation is required where noted in the criteria. Documentation may include, but not limited to, chart notes, laboratory tests, medical test results, claims records, and/or other information.'

Revised body surface area criterion phrasing from 'Patient has a body surface area of ≥ 0.39 m2' to 'Documentation provided that the patient has a body surface area of ≥ 0.39 m2.'

Rephrased 'mutation' to 'variant' for confirmation by genetic testing.

Annual Revision, Summary of Changes = No criteria changes.

1Covered Indications (HGPS)

0Covered for other progeroid conditions

1 yrApproval duration (HGPS)

≥0.39

Coverage Summary

Zokinvy (lonafarnib) is indicated to reduce mortality in Hutchinson‑Gilford Progeria Syndrome (HGPS) in patients who are at least 12 months of age and who have a body surface area of at least 0.39 m2. This Cigna coverage policy addresses prior authorization and documentation requirements for prescription benefit coverage of Zokinvy for HGPS and specifies approval criteria, specialist prescriber consultation requirements, and exclusions for other progeroid conditions.

Surface thresholds and quick facts

Minimum age≥ 12 months

Body surface area requirement≥ 0.39 m2

Approval duration1 year

Covered Indications (HGPS)1

Initial Therapy Criteria (FDA-Approved Indication)

FDA-Approved Indication: Hutchinson-Gilford Progeria Syndrome (HGPS) - Approval for 1 year

Approve for 1 year if ALL of the following are met:

Appendix reference: Refer to Appendix for acceptable LMNA variants (see Appendix Table 1 listing classic and non-classic HGPS mutations)

Appendix lists specific heterozygous mutations (e.g., c.1824C>T; p.G608G and others).

Not Medically Necessary / Exclusions

Zokinvy is considered not medically necessary for uses other than the FDA-approved HGPS indication, including but not limited to:

Processing-deficient progeroid laminopathies: Progeroid Laminopathies (other than HGPS) — efficacy not established; FDA labeling includes processing-deficient progeroid laminopathies but no clinical data demonstrating treatment effect in this population
Processing-proficient progeroid laminopathies — not indicated and would not be expected to be effective based on mechanism of action
Other progeroid syndromes: Other Progeroid Syndromes — not indicated and would not be expected to be effective based on mechanism of action

Provider Actions

Prior Authorization

Prior authorization required

Prior Authorization is required for prescription benefit coverage of Zokinvy. Approvals are provided for the duration noted (1 year for the FDA‑approved HGPS indication).

Documentation Required

Documentation required

Documentation is required where noted in the criteria. Documentation may include, but is not limited to, the following examples to support age, body surface area, genetic testing results, and specialty prescriber consultation:

Chart notes
Laboratory tests
Medical test results
Claims records

Applicable Codes

Policy identifiermixed

IP0107

Coverage Policy Number

Appendix: Genetic Variants Associated with HGPS

The Appendix lists specific heterozygous LMNA variants that are considered consistent with a diagnosis of Hutchinson‑Gilford Progeria Syndrome and may be used to confirm HGPS for coverage determinations under this policy.

Location	Mutation	Phenotype
Exon 11	c.1824C>T; p.G608G	Classic Hutchinson-Gilford Progeria Syndrome
Exon 11	c.1821G>A; p.V607V	Non-Classic Hutchinson-Gilford Progeria Syndrome
Exon 11	c.1822G>A; p.G608S	Non-Classic Hutchinson-Gilford Progeria Syndrome
Exon 11	c.1868C>G; p.T623S	Non-Classic Hutchinson-Gilford Progeria Syndrome
Intron 11	c.1968+1G>A	Non-Classic Hutchinson-Gilford Progeria Syndrome
Intron 11	c.1968+1G>C	Non-Classic Hutchinson-Gilford Progeria Syndrome
Intron 11	c.1968+2T>A	Non-Classic Hutchinson-Gilford Progeria Syndrome
Intron 11	c.1968+2T>C	Non-Classic Hutchinson-Gilford Progeria Syndrome
Intron 11	c.1968+5G>C	Non-Classic Hutchinson-Gilford Progeria Syndrome

Clinical Evidence & References

Key evidence cited in the policy includes the pivotal mortality analysis by Gordon et al. (JAMA 2018) demonstrating an association of lonafarnib treatment with reduced mortality in HGPS, and the Zokinvy prescribing information (March 2024) which provides the approved indication, dosing considerations, and supporting data.

Definitions

HGPS: Hutchinson‑Gilford Progeria Syndrome — an ultrarare, typically autosomal dominant premature aging disorder caused by heterozygous LMNA variants leading to accumulation of progerin and associated with early, severe atherosclerosis and premature mortality.

Processing‑deficient progeroid laminopathies: Progeroid laminopathies with defects in prelamin A processing that can lead to accumulation of prelamin A/progerin; FDA labeling includes these conditions but clinical efficacy of Zokinvy in these populations has not been established.

Revision History

06/01/2024annual_revision

Added documentation language: 'Documentation is required where noted in the criteria. Documentation may include, but not limited to, chart notes, laboratory tests, medical test results, claims records, and/or other information.' (Documentation requirement language added).

06/01/2024criteria_revision

Revised body surface area criterion phrasing from 'Patient has a body surface area of ≥ 0.39 m2' to 'Documentation provided that the patient has a body surface area of ≥ 0.39 m2.'

06/01/2024terminology_revision

Rephrased 'mutation' to 'variant' for confirmation by genetic testing.