CurrentMass General Brigham Health PlanPolicy N/A

Medications for Niemann-Pick Disease Type C (Aqneursa, Miplyffa)

Defines prior authorization, coverage criteria, and limits for Aqneursa (levacetylleucine) and Miplyffa (arimoclomol) for treatment of neurological manifestations of Niemann-Pick disease type C for Mass General Brigham Health Plan commercial/exchange pharmacy benefit members.

Policy Summary

PayerMass General Brigham Health Plan

PolicyMedications for Niemann-Pick Disease Type C (Aqneursa, Miplyffa)

Policy CodePolicy N/A

Change TypeNew policy

Effective DateJun 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization and submit documentation confirming NPC diagnosis, neurologic symptoms, and specialist involvement before dispensing Aqneursa or Miplyffa.

SourceLink

POLICY UPDATE CHANGES

Policy lists coverage and prior authorization criteria for Aqneursa (levacetylleucine) and Miplyffa (arimoclomol) effective 06/01/2025.

6 monthsinitial approval duration

12 monthsreauthorization duration

4/dayAqneursa daily limit

3/dayMiplyffa daily limit

15 kg

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Coverage and Medical Necessity Criteria

Initial Therapy — Authorization may be granted when the following criteria are met

Authorization may be granted when the following criteria are met:

Main eligibility: 1. Member has a diagnosis of Niemann-Pick disease type C (NPC) AND 2. Diagnosis of NPC is confirmed by at least ONE of: (a) genetically confirmed by mutations in both alleles of NPC1 or NPC2 OR (b) mutation in one allele of NPC1 or NPC2 plus either positive filipin staining or elevated cholestane triol/oxysterols (>2x ULN) AND 3. Member has at least one neurological symptom of the disease (e.g., hearing loss, vertical supranuclear gaze palsy, ataxia, dementia, dystonia, seizures, dysarthria, or dysphagia) AND 4. Requested medication is prescribed by or in consultation with a specialist knowledgeable in the treatment of NPC

Product-specific requirements: Requests for Aqneursa: member must meet ALL of the following: (i) member weighs at least 15 kilograms; (ii) requested medication will not be used with Miplyffa (arimoclomol). Requests for Miplyffa: member must meet ALL of the following: (i) member is 2 years of age or older; (ii) requested medication will not be used concomitantly with Aqneursa (levacetylleucine).

Product-specific mutually exclusive use required

Continuation Therapy — Reauthorization (Continuation of Therapy)

Reauthorization (Continuation of Therapy):

General continuation: 1. Member demonstrates a positive clinical response to therapy (e.g., slowing of disease progression, improvement in neurological symptoms of the disease).

Aqneursa continuation: Requested Aqneursa will not be used in combination with Miplyffa (arimoclomol).

Miplyffa continuation: Member's treatment regimen includes miglustat AND requested Miplyffa will not be used in combination with Aqneursa (levacetylleucine).

Concomitant use of Aqneursa (levacetylleucine) and Miplyffa (arimoclomol) is prohibited. Each product’s coverage criteria explicitly state the medication must not be used in combination with the other; authorization for one product will not be granted if the member is receiving the other.

Requests from members who are new to the plan within the past 90 days are eligible for authorization only if the member is currently receiving the requested medication. Continuity is not established for medications that were obtained only as samples or through a manufacturer patient assistance program.

Initial Therapy

Initial therapy — initial authorization criteria (detailed nested criteria for each product)

Initial authorization criteria

Diagnostic and clinical criteria: Confirmed NPC diagnosis (genetically confirmed by mutations in both alleles of NPC1 or NPC2, or mutation in one allele plus positive filipin staining or elevated cholestane triol/oxysterols >2x ULN) AND at least one neurological symptom AND medication prescribed by or in consultation with a specialist knowledgeable in NPC.

Product-specific: Aqneursa (levacetylleucine): member must weigh at least 15 kilograms AND Aqneursa will not be used concomitantly with Miplyffa. Miplyffa (arimoclomol): member must be >= 2 years of age AND Miplyffa will not be used concomitantly with Aqneursa.

Product-specific mutually exclusive use required

Continuation of Therapy

Continuation of therapy — reauthorization requirements (clinical response, documentation of benefit, renewal periods)

Reauthorization requirements

General: Member demonstrates a positive clinical response to therapy (e.g., slowing of disease progression, improvement in neurological symptoms).

Aqneursa: Requested Aqneursa must not be used in combination with Miplyffa (arimoclomol).

Miplyffa: Member's treatment regimen must include miglustat AND requested Miplyffa must not be used in combination with Aqneursa (levacetylleucine).

Reauthorizations are granted per policy renewal intervals

Key Eligibility Metrics

Aqneursa minimum weight

Minimum weight>= 15 kilograms

Applicable productAqneursa (levacetylleucine)

PurposeRequired for initial authorization of Aqneursa

Miplyffa minimum age

Minimum age>= 2 years

Applicable productMiplyffa (arimoclomol)

PurposeRequired for initial authorization of Miplyffa

Product Quantity Limits

Aqneursa (levacetylleucine) packets — daily quantity limit

Daily quantity limit4 packets per day

Drug formulationAqneursa (levacetylleucine) packets

Applies toInitial and reauthorization approvals as enforced by policy

Miplyffa (arimoclomol) capsules — daily quantity limit

Daily quantity limit3 capsules per day

Drug formulationMiplyffa (arimoclomol) capsules

Applies toInitial and reauthorization approvals as enforced by policy

Authorization, Documentation, and Dispensing Requirements

Prior Authorization

Prior authorization required for Aqneursa and Miplyffa

Prior authorization is required for Aqneursa (levacetylleucine) and Miplyffa (arimoclomol) under the pharmacy benefit; these products are designated specialty medications and must be dispensed at a contracted specialty pharmacy. Initial approvals are for 6 months and reauthorizations for 12 months.

Benefit: Pharmacy Benefit; Program Type: Prior Authorization
Initial approval duration: 6 months; Reauthorization duration: 12 months
Must be filled at a contracted specialty pharmacy

Step Therapy

No step therapy requirement

No step therapy requirement is specified in this policy for Aqneursa or Miplyffa.

Dispensing and Specialty Pharmacy

Billing Rule

Dispense at contracted specialty pharmacy

These medications have been designated specialty and must be dispensed at a contracted specialty pharmacy under the pharmacy benefit.

Contact Pharmacy Benefit: Phone 800-711-4555; Fax 844-403-1029

Diagnostic Confirmation and Definitions

Confirmed NPC — Diagnostic confirmation criteria

DefinitionDiagnostic confirmation for NPC may be genetic (mutations in both alleles of NPC1 or NPC2)

Alternate confirmationMutation in one allele plus positive filipin staining

Alternate confirmation 2Mutation in one allele plus elevated cholestane triol/oxysterols (>2x ULN)

Background

Niemann-Pick disease type C (NPC) is a genetic, neurodegenerative disorder characterized by progressive neurological dysfunction. Two recently addressed therapies in this policy are Aqneursa (levacetylleucine) and Miplyffa (arimoclomol), each indicated for treatment of neurological manifestations of NPC under product-specific age/weight and regimen requirements. Authorization requires a confirmed diagnosis of NPC (genetic confirmation or genetic plus laboratory/histochemical evidence), at least one neurological symptom, and management by or consultation with a specialist knowledgeable in NPC. Product-specific limits include a minimum weight requirement for Aqneursa (see coding thresholds) and a minimum age plus requirement for concomitant miglustat use with Miplyffa.

Policy Revision History

2025-03-12policy_created_and_reviewed

New clinical policy created and reviewed at the March Pharmacy & Therapeutics (P&T) meeting.

2025-06-01coverage_and_prior_auth_effective_dateLatest

Policy listing coverage and prior authorization criteria for Aqneursa (levacetylleucine) and Miplyffa (arimoclomol) became effective.