Currentmedical mutual - ohioPolicy N/A

Aqneursa (levacetylleucine) for Niemann-Pick disease type C

Pharmacy benefit prior authorization guidance for Aqneursa (levacetylleucine oral suspension) for treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in patients meeting specified diagnostic, age/weight, prescriber, and concomitant therapy criteria; includes initial and continuation approval durations and conditions not recommended for approval.

Policy Summary

Payermedical mutual - ohio

PolicyAqneursa (levacetylleucine) for Niemann-Pick disease type C

Policy CodePolicy N/A

Change TypeAnnual review — no material change

Effective Date

Next Review DateAug 21, 2025

Key ActionPrior authorization is recommended; approve only when the listed criteria are met and require specialist prescribing/consultation and genetic documentation for initial approval.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes noted in this update.

1Covered Indication(s)

3Not Recommended Conditions

2Approval Durations

Coverage Summary

Coverage stance: Aqneursa (levacetylleucine) is covered with criteria for the treatment of neurological manifestations of Niemann-Pick disease type C when patients meet the policy's specified diagnostic, age/weight, prescriber, and concomitant therapy criteria. The scope is pharmacy benefit prior authorization guidance for Aqneursa (levacetylleucine oral suspension) including initial and continuation approval durations and conditions not recommended for approval.

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Initial Therapy Criteria

Recommended Authorization Criteria - Niemann-Pick disease type C

Coverage of Aqneursa is recommended in those who meet the following criteria:

A) Initial Therapy - Approve for 6 months if ALL of the following are met

Patient is ≥ 4 years of age>= 4 years
Patient weighs ≥ 15 kg>= 15 kg
Patient has one or more neurologic symptom(s) of Niemann-Pick disease type C
Examples: loss of motor function, difficulty swallowing, speech and cognitive impairment
Diagnosis established by a genetic test showing biallelic pathogenic variants in either the NPC1 gene or NPC2 genebiallelic pathogenic variants
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
Initial approval requires specialist involvement
ONE of
- Aqneursa is prescribed in combination with miglustat
- History of failure, contraindication, or intolerance to miglustat

B) Patient Currently Receiving Aqneursa - Approve for 1 year if BOTH (i and ii) are met (and iii)

According to the prescriber, patient has derived benefit from treatment defined as disease stabilization, slowed progression, or improvement
Benefit defined as disease stabilization, slowed progression, or improvement
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
ONE of
- Aqneursa is prescribed in combination with miglustat
- History of failure, contraindication, or intolerance to miglustat

Continuation Therapy Criteria

Recommended Authorization Criteria - Niemann-Pick disease type C

Coverage of Aqneursa is recommended in those who meet the following criteria:

B) Patient Currently Receiving Aqneursa - Approve for 1 year if BOTH (i and ii) are met (and iii)

(i): According to the prescriber, patient has derived benefit from treatment defined as disease stabilization, slowed progression, or improvement
Benefit from treatment defined as disease stabilization, slowed progression, or improvement
(ii): 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
(iii) ONE of
- Aqneursa is prescribed in combination with miglustat
- History of failure, contraindication, or intolerance to miglustat

Conditions Not Recommended for Approval

Listed conditions where Aqneursa is not recommended for coverage due to lack of supportive evidence or potential safety concerns:

Ataxia-Telangiectasia (insufficient data; Phase II study underway with results pending)
Combination use with Miplyffa (arimoclomol capsules) — no data available regarding combination use
GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) — preliminary/limited data; additional study needed
Circumstances not listed in the Recommended Authorization Criteria

Provider Actions

Prior Authorization

Prior authorization recommended

Prior authorization is recommended for pharmacy benefit coverage of Aqneursa; approval is recommended only when criteria are met. The company may request additional documentation and may deny reimbursement if the drug or services are not medically necessary, investigational, or otherwise outside scope of benefits.

The Company may request additional documentation (patient records, test results, provider credentials) to support medical necessity.
Reimbursement may be denied if the drug is determined to be not medically necessary, investigational, experimental, or outside benefit scope.

Documentation Required

Prescriber specialty requirement

Initial approval requires the medication be prescribed by or in consultation with a specialist experienced in NPC: a geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or a physician who specializes in the treatment of Niemann-Pick disease type C or related disorders.

Accepted specialties: geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or physician specializing in Niemann-Pick disease type C or related disorders

Documentation Required

Genetic test documentation

For initial therapy, the diagnosis must be established by a genetic test showing biallelic pathogenic variants in either the NPC1 gene or NPC2 gene; documentation of this genetic testing is required.

Requirement: document biallelic pathogenic variants in NPC1 or NPC2

Documentation Required

Continuation response requirement

For patients currently receiving Aqneursa, the prescriber must attest that the patient has derived benefit from treatment, defined as disease stabilization, slowed progression, or improvement, in order to approve extended therapy.

Prescriber must attest benefit defined as disease stabilization, slowed progression, or improvement

Background

Background: Aqneursa (levacetylleucine) is indicated for treatment of neurological manifestations of Niemann-Pick disease type C in patients weighing ≥ 15 kg. Because of specialized evaluation and monitoring needs, initial approval requires prescription by or consultation with a specialist experienced in NPC (geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or physician specializing in NPC). The policy references pivotal and supportive literature and the prescribing information to support the indication and criteria.

Policy Summary

Payermedical mutual - ohio

PolicyAqneursa (levacetylleucine) for Niemann-Pick disease type C

Policy CodePolicy N/A

Change TypeAnnual review — no material change

Effective Date

Next Review DateAug 21, 2025

Key ActionPrior authorization is recommended; approve only when the listed criteria are met and require specialist prescribing/consultation and genetic documentation for initial approval.

SourceLink

On This Page

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Aqneursa (levacetylleucine) for Niemann-Pick disease type C

Coverage Summary

Initial Therapy Criteria

Continuation Therapy Criteria

Conditions Not Recommended for Approval

Applicable Codes

Provider Actions

Clinical Evidence & References

Background

Revision History