CurrentUnitedHealthcarePolicy 2025 P 1461-2

Aqneursa (levacetylleucine) prior authorization

Criteria and prior authorization rules for coverage of Aqneursa (levacetylleucine) to treat neurological manifestations of Niemann-Pick disease type C (NPC) in eligible patients; affects providers requesting pharmacy benefit coverage under UnitedHealthcare.

Policy Summary

PayerUnitedHealthcare

PolicyAqneursa (levacetylleucine) prior authorization

Policy CodePolicy 2025 P 1461-2

Change TypeNew prior authorization programmaterial additions

Effective DateApr 1, 2025

Next Review Date

Key ActionObtain prior authorization documenting NPC diagnosis and that the patient is not receiving Aqneursa with Miplyffa; authorization issued for 12 months.

SourceLink

POLICY UPDATE CHANGES

New prior authorization program established for Aqneursa (levacetylleucine).

Criteria added that Aqneursa not be taken in combination with Miplyffa (arimoclomol).

2025-04-01policy effective date

12 monthsauthorization duration

≥15 kgpediatric weight threshold

11/2024, 1/2025P&T approval dates

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Coverage Criteria for Aqneursa (levacetylleucine)

Initial Authorization

Covered when ALL of the following are met:

Initial authorization criteria: (a) Diagnosis of Niemann-Pick disease type C (NPC); (b) Aqneursa is being used to treat neurological manifestations of NPC; (c) Patient is not receiving Aqneursa in combination with Miplyffa (arimoclomol)

Authorization issued for 12 months.

Reauthorization

Covered when ALL of the following are met:

Reauthorization criteria: (a) Documentation of positive clinical response to Aqneursa (e.g., slowed disease progression from baseline based on assessment with NPC-specific scales); (b) Patient is not receiving Aqneursa in combination with Miplyffa (arimoclomol)

Authorization issued for 12 months.

Concurrent use of Aqneursa (levacetylleucine) with Miplyffa (arimoclomol) is explicitly excluded from approval; requests will be denied if the patient is prescribed both agents concurrently.

Initial Therapy

Initial coverage criteria

Initial: Diagnosis of NPC; used to treat neurological manifestations; not used with Miplyffa (arimoclomol)

Authorization issued for 12 months.

Continuation / Reauthorization Criteria

Continuation/Reauthorization

Reauthorization/continuation coverage rules

Reauthorization: Documentation of positive clinical response using NPC-specific scales; no concurrent Miplyffa (arimoclomol)

12-month authorization

Coding and Patient Characteristics

Pediatric weight threshold

Pediatric weight thresholdPatients must weigh ≥15 kg

Applicable populationAdults and pediatric patients meeting the weight threshold

Source indicationIndication limited to neurological manifestations of Niemann-Pick disease type C (NPC)

Provider Actions and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior Authorization Required: Prior authorization is required for initiation of Aqneursa (for NPC). Approvals for initial requests will be issued for 12 months.

Diagnosis must be Niemann-Pick disease type C (NPC).
Aqneursa must be used to treat neurological manifestations of NPC.
Patient must not be receiving Aqneursa in combination with Miplyffa (arimoclomol).

Documentation Required

Reauthorization Needed: Document Continued Benefit

Reauthorization Requirements: Reauthorization is required every 12 months and approval is contingent on documentation of clinical benefit.

Documentation of positive clinical response to Aqneursa (e.g., slowed disease progression from baseline using NPC-specific assessment scales).
Patient must not be receiving Aqneursa in combination with Miplyffa (arimoclomol).
Authorization will be issued for 12 months on reauthorization.

Note

Automated Approval Possible; Supply Limits May Apply

Automated Approval and Supply Limits: UnitedHealthcare may use automated approval or re-approval based on prior claims, medication history, diagnosis codes (ICD-10), and claim logic. Supply limits may apply.

Automated approval processes vary by program and therapeutic class and may allow approval without full chart documentation when previous claim/medication history supports use.
Supply limits may be in place — check benefit-specific quantity limits and plan rules.

Denial Risk

Denial Triggers — What to Avoid/Omit

Denial Triggers and Required Documentation: Denial may be issued if required documentation or conditions are missing.

Concurrent use with Miplyffa (arimoclomol) is a trigger for denial.
Missing diagnosis of NPC or lack of documentation that Aqneursa is being used for neurological manifestations may result in denial.
For reauthorization, insufficient documentation of positive clinical response (e.g., no NPC-specific scale assessments or no evidence of slowed progression from baseline) may prompt denial.

Background

Aqneursa (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients (policy notes specify pediatric use in patients weighing >=15 kg). The drug’s package insert and the prior authorization program establish that coverage is limited to this indication and that reauthorization requires documentation of a positive clinical response, such as slowed disease progression measured with NPC-specific assessment scales.

Definitions

Neurological manifestations of NPC

DefinitionNeurological manifestations of NPC: clinical features in patients with Niemann-Pick disease type C that affect the nervous system and are the target of Aqneursa therapy

Clinical scopeIncludes neurologic signs and symptoms attributed to NPC (as target of therapy)

Treatment relevanceAqneursa is indicated to treat these neurological manifestations in eligible patients

Step Therapy / Automated Approval Rules

Automated approval logic	What may trigger automated approval	Notes / limits
UnitedHealthcare may approve initial and re-authorization requests based solely on previous claim/medication history, diagnosis codes (ICD-10), and/or claim logic.	Prior claims showing prior dispensing of Aqneursa, relevant NPC diagnosis codes, or other claim-based rules may be used to trigger automated approval or re‑approval.	Use of automated approval and re-approval processes varies by program and therapeutic class; supply limits may be in place.

Quantity Limits

Aqneursa (levacetylleucine) — supply limits

Quantity/supply limitsSupply limits may be in place for Aqneursa (levacetylleucine)

Automated approvalsUnitedHealthcare may use previous claim/medication history and diagnosis codes for automated approval or re-approval, which may interact with supply limits

Program variabilityUse of automated approval and supply-limit policies may vary by program or therapeutic class

Site of Care

Note

Infusion center — no site restrictions listed

No site‑of‑care or infusion‑center specific restrictions are listed in the program. UnitedHealthcare may, however, consider prior claims/medication history and diagnosis codes when applying automated approval or re‑approval processes.

Policy Summary

PayerUnitedHealthcare

PolicyAqneursa (levacetylleucine) prior authorization

Policy CodePolicy 2025 P 1461-2

Change TypeNew prior authorization programmaterial additions

Effective DateApr 1, 2025

Next Review Date

Key ActionObtain prior authorization documenting NPC diagnosis and that the patient is not receiving Aqneursa with Miplyffa; authorization issued for 12 months.

SourceLink

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