CurrentMass General Brigham Health PlanPolicy N/A

Medications for Treatment of Niemann-Pick Disease Type C (Aqneursa, Miplyffa) - Prior Authorization and Coverage Criteria

This policy governs prior authorization, quantity limits, specialty pharmacy designation, initial and continuation coverage criteria for Aqneursa (levacetylleucine) and Miplyffa (arimoclomol) for Mass General Brigham Health Plan members.

Policy Summary

PayerMass General Brigham Health Plan

PolicyMedications for Treatment of Niemann-Pick Disease Type C (Aqneursa, Miplyffa) - Prior Authorization and Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJun 1, 2025

Next Review DateMar 12, 2025

Key ActionObtain prior authorization and document genetically confirmed diagnosis of Niemann-Pick disease type C with required specialist involvement.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsInitial approval duration

12 monthsReauthorization duration

>=15 kgAqneursa minimum weight

>=2 yrsMiplyffa minimum age

Aqneursa: 4 p/day

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Coverage Criteria

Initial Therapy

Authorization may be granted when the following criteria are met:

Initial approval general criteria: Member has a diagnosis of Niemann-Pick disease type C (NPC); diagnosis is genetically confirmed by mutations in both alleles of NPC1 or NPC2 OR mutation in one allele plus corroborating testing (positive filipin staining or elevated cholestane triol/oxysterols >2x ULN); member has at least one neurological symptom of NPC (e.g., hearing loss, vertical supranuclear gaze palsy, ataxia, dementia, dystonia, seizures, dysarthria, or dysphagia); requested medication is prescribed by or in consultation with a specialist knowledgeable in the treatment of NPC.

Product-specific eligibility

Aqneursa-specific: Member weighs at least 15 kilograms; requested medication will not be used with Miplyffa (arimoclomol).
Miplyffa-specific: Member is at least 2 years of age; member's treatment regimen will include miglustat; requested medication will not be used with Aqneursa (levacetylleucine).

New-to-plan continuity: Authorization may also be granted for members new to the plan within the past 90 days who are currently receiving the requested medication (excludes samples or manufacturer patient assistance programs).

Continuation Therapy

Reauthorization will be approved when the following are met:

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

Product-specific continuation: For Aqneursa: requested medication must not be used in combination with Miplyffa. For Miplyffa: member's regimen includes miglustat and requested medication must not be used in combination with Aqneursa.

Authorization for Aqneursa (levacetylleucine) or Miplyffa (arimoclomol) requires that the requested medication will not be used concurrently with the other agent. Each request must explicitly confirm that the member will not receive Aqneursa and Miplyffa together (concurrent use is prohibited).

Additional coverage-level notes: prior authorization is required for these specialty medications and may be granted for members new to the plan within the past 90 days who are currently receiving the medication (excludes samples and manufacturer patient assistance). Documentation must demonstrate genetic confirmation of Niemann-Pick disease type C and specialist involvement as specified in the criteria. Requests may be denied if genetic confirmation, specialist consultation, age/weight thresholds, or the non-concurrent use requirement are not met.

Coding and Thresholds

Age/Weight thresholds — Aqneursa and Miplyffa

Aqneursa (levacetylleucine) weight thresholdPatient must weigh at least 15 kilograms

Miplyffa (arimoclomol) age thresholdPatient must be 2 years of age or older

Concurrent use restrictionRequested medication must not be used in combination with the other NPC medication (Aqneursa and Miplyffa cannot be used together)

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for specialty medications and for members new to the plan within the past 90 days who are currently receiving the requested medication (excludes samples or manufacturer patient assistance program supplies). The medication is designated specialty and must be filled at a contracted specialty pharmacy.

Specialty fill required at contracted specialty pharmacy
New-to-plan continuity allowed within 90 days (excludes samples / patient assistance)

Step Therapy

No step therapy requirements are specified for these medications.

Background

Niemann-Pick disease type C (NPC) is a genetic, progressive neurodegenerative disorder characterized by neurological manifestations such as vertical supranuclear gaze palsy, ataxia, dystonia, seizures, hearing loss, dysarthria, dysphagia, and cognitive decline. Diagnosis for coverage purposes must be genetically confirmed by mutations in both alleles of NPC1 or NPC2, or by a mutation in one allele plus corroborating laboratory evidence (positive filipin staining or elevated cholestane triol/oxysterols > 2× ULN).

Therapeutic context: Aqneursa (levacetylleucine) is indicated for treatment of neurological manifestations of NPC in adults and pediatric patients who weigh at least 15 kg. Miplyffa (arimoclomol) is indicated for use in combination with miglustat for treatment of neurological manifestations of NPC in patients aged 2 years and older. Both agents must be prescribed by or in consultation with a specialist knowledgeable in NPC management.

Definitions

Miplyffa (arimoclomol) — indication and age-related note

IndicationMiplyffa (arimoclomol) is indicated for use in combination with miglustat for treatment of neurological manifestations of Niemann‑Pick disease type C (NPC)

Age requirementIndicated for adults and pediatric patients 2 years of age and older

Therapy combination requirementTreatment regimen must include miglustat when prescribing Miplyffa

Aqneursa (levacetylleucine) — indication and weight-related note

IndicationAqneursa (levacetylleucine) is indicated for treatment of neurological manifestations of Niemann‑Pick disease type C (NPC)

Weight requirementIndicated for adults and pediatric patients weighing at least 15 kilograms

Policy Summary

PayerMass General Brigham Health Plan

PolicyMedications for Treatment of Niemann-Pick Disease Type C (Aqneursa, Miplyffa) - Prior Authorization and Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJun 1, 2025

Next Review DateMar 12, 2025

Key ActionObtain prior authorization and document genetically confirmed diagnosis of Niemann-Pick disease type C with required specialist involvement.

SourceLink

On This Page

Documentation Required

Required Documentation

Documentation must include genetic confirmation and clinical evidence of diagnosis. Acceptable documentation: genetic testing showing pathogenic mutations in both alleles of NPC1 or NPC2, or mutation in one allele plus supportive laboratory or histologic evidence (positive filipin staining or elevated cholestane triol/oxysterols >2× ULN). Also include documentation of diagnosis, member age/weight where applicable, and that the prescribing clinician is a specialist knowledgeable in NPC care.

Genetic confirmation: mutations in both alleles of NPC1 or NPC2
If mutation in only one allele: require positive filipin staining or elevated cholestane triol/oxysterols (>2× ULN)
Documentation of at least one neurological symptom (e.g., hearing loss, vertical supranuclear gaze palsy, ataxia, dementia, dystonia, seizures, dysarthria, dysphagia)
Prescriber is or consulted with a specialist experienced in NPC
For Aqneursa: member weight ≥ 15 kg; for Miplyffa: member ≥ 2 years and regimen includes miglustat

Denial Risk

Triggers for Denial

Requests may be denied if required confirmation or documentation is missing, if the diagnosis is not supported by genetic or corroborative testing, if the prescriber is not a specialist or there is no documented specialist consultation, if age/weight criteria are not met, or if a requested agent is prescribed concomitantly with a disallowed agent (Aqneursa with Miplyffa or vice versa).

Missing genetic confirmation or required supportive testing
No documentation of at least one NPC neurological symptom
No specialist prescriber or lack of documented specialist consultation
Aqneursa: member weight < 15 kg
Miplyffa: member age < 2 years or treatment regimen does not include miglustat
Concurrent use of Aqneursa and Miplyffa (not allowed)

Concurrent use restrictionRequested Aqneursa must not be used in combination with Miplyffa (arimoclomol)