CurrentAvMedPolicy N/A

Miplyffa

Prior authorization form and coverage criteria for Miplyffa (arimoclomol) for treatment of Niemann-Pick disease type C (NPC), including initial and reauthorization clinical requirements, dosing guidance, quantity limits, specialty pharmacy, and documentation requirements.

Policy Summary

PayerAvMed

PolicyMiplyffa

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionPrescribing physician must sign and print name on the request and fax the completed form to 1-305-671-0200; submit genetic testing and baseline neurologic assessment documentation and verify concomitant miglustat use.

SourceLink

POLICY UPDATE CHANGES

No material changes to clinical criteria or coverage — policy remains unchanged.

1Specialty Pharmacy designated

90/30dQuantity limit

12 moAuth duration (init/reauth)

Coverage Summary

This policy covers Miplyffa (arimoclomol) for treatment of Niemann-Pick disease type C (NPC) with prior authorization and clinical criteria. Coverage is conditional (covered_with_criteria) requiring fulfillment of the specified initial or reauthorization criteria. Dispensing is via Specialty Pharmacy and approvals are issued for a 12-month authorization period. A quantity limit of 90 capsules per 30 days applies (one bottle per 30 days).

Quick Facts / Key Thresholds

Age>= 2 years

Cholestane-triol threshold> 2 times the upper limit of normal

Quantity limit90 capsules (1 bottle) per 30 days, all strengths

Initial / Reauthorization duration12 months

Specialty PharmacyProprium Rx

Policy Summary

PayerAvMed

PolicyMiplyffa

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

SourceLink

On This Page

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Initial Therapy Criteria

Initial Authorization / Medically Necessary

All criteria must be met for approval. To support each line checked, documentation (labs, diagnostics, chart notes) must be provided.

ALL of the following

Age: Member is >= 2 years of age
Prescriber specialty: 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
Diagnosis confirmation (ONE of)
- Biallelic variants: Biallelic pathogenic variants in either the NPC1 gene or NPC2 gene
  submit documentation
- Single allele plus test: Mutations in only one allele of NPC1 or NPC2 plus either positive filipin staining or elevated cholestane-triol level (>2 times the upper limit of normal)> 2 times the upper limit of normal
  submit documentation
Neurologic symptoms: Member has at least ONE neurological symptom(s) of Niemann-Pick disease type C (e.g., loss of motor function, swallowing, and speech and cognitive impairment)
submit documentation
Ambulation: Member can walk independently or with assistance
Baseline assessment: Provider must submit a baseline assessment scale documenting current NPC neurologic symptom(s)
submit documentation
Concomitant miglustat: Requested medication will be taken in combination with miglustat
verified by pharmacy paid claims
Prohibited concomitant therapy: Requested medication will NOT be used in combination with Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C

Continuation Therapy Criteria

Reauthorization / Continued Therapy

All criteria must be met for approval. To support each line checked, documentation (labs, diagnostics, chart notes) must be provided.

Reauthorization criteria

Continued initial criteria: Member continues to meet ALL initial authorization criteria
Concomitant miglustat: Requested medication will be taken in combination with miglustat
verified by pharmacy paid claims
Evidence of benefit: Member has derived benefit from treatment defined as disease stabilization, slowed progression, or improvement, according to the prescriber

Provider Actions & Requirements

Prior Authorization

Submit signed prior authorization form

The prescribing physician must sign and clearly print their name on the prior authorization request. Fax the completed form to 1-305-671-0200. Incomplete, incorrect, or illegible information may delay the authorization process.

Documentation Required

Provide diagnostic/genetic documentation

Submit documentation of genetic testing to confirm NPC (either biallelic pathogenic variants in NPC1 or NPC2, or a single allele mutation plus positive filipin staining or elevated cholestane-triol >2× ULN). Also submit a baseline neurologic assessment scale and evidence of at least one neurologic symptom of NPC.

Documentation Required

Verify concomitant miglustat use

Concomitant use with miglustat must be verified; this verification is performed via pharmacy paid claims.

Denial Risk

Concomitant use with Aqneursa (levacetylleucine) prohibited

Requests will be denied if the medication is used in combination with Aqneursa (levacetylleucine) for treatment of the neurological manifestations of Niemann-Pick disease type C.

Billing Rule

Specialty pharmacy dispensing

This medication is dispensed through a specialty pharmacy: Proprium Rx.

Documentation Required

Samples do not meet criteria

Use of samples to initiate therapy does not meet step edit or preauthorization criteria and therefore does not satisfy initiation requirements.

Background

This form documents the prior authorization and clinical criteria required to approve Miplyffa (arimoclomol) for Niemann-Pick disease type C (NPC). Miplyffa is the brand name for arimoclomol used for NPC. Approval requires genetic confirmation of NPC (either biallelic pathogenic variants in NPC1 or NPC2, or a single allele mutation plus either positive filipin staining or an elevated cholestane-triol level > 2 times the upper limit of normal), evidence of at least one neurologic symptom of NPC, a baseline neurologic assessment, and prescribing by or in consultation with an appropriate specialist. Concomitant use with miglustat is required (verified by pharmacy paid claims). Use of Miplyffa in combination with Aqneursa (levacetylleucine) for treatment of NPC neurological manifestations is prohibited.

OpenPayer

Miplyffa

Coverage Summary

Initial Therapy Criteria

Continuation Therapy Criteria

Provider Actions & Requirements

Applicable Codes & Limits

Background

Clinical Evidence

Revision History