CurrentCignaPolicy IP0715

Aqneursa (levacetylleucine) for Niemann-Pick disease type C

Defines prior authorization and coverage criteria for Aqneursa (levacetylleucine oral suspension) to treat neurological manifestations of Niemann-Pick disease type C for members of Cigna-administered health benefit plans.

Policy Summary

PayerCigna

PolicyAqneursa (levacetylleucine) for Niemann-Pick disease type C

Policy CodePolicy IP0715

Change TypeNew policy

Effective DateDec 15, 2025

Next Review DateDec 15, 2025

Key ActionSubmit prior authorization; approvals are provided for 6 months initially or 1 year for patients already receiving Aqneursa.

SourceLink

POLICY UPDATE CHANGES

New policy established for coverage of Aqneursa.

≥4 yrsminimum age for initial therapy

≥15 kgminimum weight for eligibility

6 monthsinitial approval

1 yearcontinuation approval

n=60pivotal trial enrollment

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Coverage Criteria for Aqneursa (levacetylleucine)

Initial Therapy — Approve for 6 months

Approve for 6 months if the patient meets ALL of the following (A.i–v):

Initial Therapy for NPC: (i) Patient is ≥ 4 years of age.

Initial Therapy for NPC: (ii) Patient weighs ≥ 15 kg.

Initial Therapy for NPC: (iii) Patient has one or more neurologic symptom(s) of Niemann-Pick disease type C (documentation required).

Examples of neurologic symptoms: loss of motor function, difficulty swallowing, speech and cognitive impairment.

Initial Therapy for NPC: (iv) Diagnosis is established by a genetic test showing biallelic pathogenic variants in either the NPC1 gene or NPC2 gene (documentation required).

Initial Therapy for NPC: (v) Medication is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or a physician who specializes in the treatment of Niemann-Pick disease type C or related disorders.

Patient is Currently Receiving Aqneursa — Approve for 1 year

Approve for 1 year if the patient meets BOTH of the following (B.i–ii):

Currently Receiving Aqneursa: (i) According to the prescriber, patient has derived benefit from treatment defined as disease stabilization, slowed progression, or improvement.

Prescriber documentation/attestation of benefit is required.

Currently Receiving Aqneursa: (ii) The medication is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or a physician who specializes in the treatment of Niemann-Pick disease type C or related disorders.

The following uses of Aqneursa are considered experimental, investigational, or unproven and are not covered: (1) treatment of Ataxia‑Telangiectasia (clinical trials are ongoing and results are not available), (2) combination use with Miplyffa (arimoclomol) (no data on combined use with Aqneursa), and (3) treatment of GM2 gangliosidoses (Tay‑Sachs and Sandhoff diseases) despite limited Phase IIb data; additional study is needed. This list may not be exhaustive and will be updated as new evidence becomes available.

Use of Aqneursa for any indication not listed in the FDA‑approved indication section is considered experimental, investigational, or unproven and therefore not covered under this policy.

Provider Actions and Prior Authorization

Prior Authorization

Prior authorization required — approvals for specified durations

Prior authorization is required for benefit coverage of Aqneursa; approvals are provided for 6 months for initial therapy and 1 year for patients currently receiving Aqneursa. Approvals require the medication be prescribed by or in consultation with a specialist as specified in the criteria. (1 month = 30 days when durations are authorized in months.)

Initial therapy approval duration: 6 months (meets ALL A.i–v)
Continuation/current therapy approval duration: 1 year (meets BOTH B.i–ii)

Step Therapy

Step therapy — none specified

No step therapy (prior medication trial) requirements are specified in this policy for Aqneursa.

Definitions and Measurement Scales

Genetically confirmed NPC

DefinitionDiagnosis established by genetic testing showing biallelic pathogenic variants in either NPC1 or NPC2; identification of two disease‑causing alleles confirms NPC.

Required testMutation analysis of NPC1 and NPC2 genes is mandatory to confirm the diagnosis; genetic testing is the preferred method for prenatal diagnosis and carrier identification.

Interpretation noteInterpretation of novel missense or splicing variants requires caution and verification of pathogenicity; presence of very severe mutations (frameshift, nonsense, large deletion) in both alleles often results in early infantile neurological disease.

Epidemiology relevanceNPC is caused by mutations in NPC1 (≈90–95%) or NPC2 (≈5%); around 700 NPC1 variants reported with ~420 considered pathogenic; ~26 pathogenic NPC2 mutations described.

Most actionable requirementDocumentation of genetic test results showing two disease‑causing alleles (biallelic pathogenic variants in NPC1 or NPC2) is required for coverage of initial therapy.

Background

Niemann‑Pick disease type C (NPC) is an autosomal recessive, progressive lysosomal storage disorder characterized by accumulation of lipids in the brain, liver, and spleen leading to heterogeneous neurologic, visceral, and psychiatric manifestations. NPC is an ultra‑rare condition with variable age of neurologic onset and progressive deficits affecting motor function, swallowing, speech, and cognition. Aqneursa is indicated for the neurological manifestations of NPC in patients meeting the policy’s age and weight thresholds.

Revision History

2025-12-15Annual revisionLatest

Annual revision — no clinical criteria changes noted; policy effective date maintained at 2025-12-15.

2025-03-15Policy establishment

New policy established for coverage of Aqneursa (levacetylleucine) — initial creation of policy document.

Policy Summary

PayerCigna

PolicyAqneursa (levacetylleucine) for Niemann-Pick disease type C

Policy CodePolicy IP0715

Change TypeNew policy

Effective DateDec 15, 2025

Next Review DateDec 15, 2025

Key ActionSubmit prior authorization; approvals are provided for 6 months initially or 1 year for patients already receiving Aqneursa.

SourceLink

On This Page

Documentation Required

Required documentation — genetic and clinical evidence

Documentation is required where noted in the coverage criteria and may include chart notes, laboratory tests, claims records, and other information; genetic test documentation showing biallelic pathogenic variants in NPC1 or NPC2 and documentation of neurologic symptoms are specifically required by the criteria.

Acceptable documentation may include chart notes, laboratory tests, and claims records.
Genetic test showing biallelic pathogenic variants in NPC1 or NPC2 is required for initial therapy.
Documentation of one or more neurologic symptoms is required for initial therapy.

Denial Risk

Triggers for denial — failure to meet criteria or non‑covered indications

Requests that do not meet the specified criteria may be denied — examples include lack of genetic confirmation of biallelic NPC1 or NPC2 pathogenic variants, patient below the age (≥ 4 years) or weight (≥ 15 kg) thresholds, or requests for uses listed as not covered (e.g., Ataxia‑Telangiectasia, combination use with Miplyffa, GM2 gangliosidoses).

Lack of required genetic test documentation (biallelic NPC1/NPC2) can trigger denial.
Requests for indications considered experimental/investigational (Conditions Not Covered) can be denied.

SARA / mSARA

SARA overviewSARA is an eight‑item clinical rating scale (gait, stance, sitting, speech disturbance, finger‑chase, nose‑to‑finger, fast alternating hand movements, heel‑along‑shin); scores range 0–40 with lower scores indicating better neurologic status.

mSARA overviewmSARA is the modified SARA that excludes the sitting and stance domains; scores range 0–30 with lower scores indicating better neurologic status.

Clinical significanceA 1‑point change in the SARA total score is considered a clinically meaningful transition (≥ -1 point improvement or ≥ +1 point worsening).

Use in trialsPrimary efficacy endpoint outside the US used SARA total score at 12 weeks; in the US the primary endpoint was mSARA.

Baseline referenceMean baseline SARA was 15.88 and mean baseline mSARA was 13.20 in the pivotal trial cohort.