CurrentCignaPolicy N/A

Opfolda (miglustat) for Pompe disease

Prior authorization policy describing coverage criteria for Opfolda (miglustat capsules) as enzyme stabilizer therapy in combination with Pombiliti for late-onset Pompe disease in adults >40 kg, including required diagnostic evidence, prescriber specialty, duration of approval, and non-covered indications.

Policy Summary

PayerCigna

PolicyOpfolda (miglustat) for Pompe disease

Policy CodePolicy N/A

Change TypeClarified; Annual revision

Effective Date

Next Review Date

Key ActionPrior authorization is recommended; approvals provided for 1 year.

POLICY UPDATE CHANGES

Genetic testing language rephrased to specify 'biallelic pathogenic or likely pathogenic acid alpha-glucosidase gene variants'.

Annual revision on 05/07/2025 noted; 'No criteria changes' for that revision.

1Covered Indication (FDA-approved)

1Not Covered Example (Gaucher disease)

1 yearApproval Duration

Coverage Summary

Prior authorization policy describing coverage criteria for Opfolda (miglustat capsules) as an enzyme stabilizer to be used in combination with Pombiliti for late-onset Pompe disease (acid alpha-glucosidase deficiency) in adults weighing > 40 kg. Approvals are provided for 1 year. The policy requires diagnostic evidence and prescriber specialty involvement and specifies that Opfolda has no monotherapy activity for Pompe disease; indications other than the FDA-approved use (for example, Gaucher disease) are not covered.

FDA-Approved Indication - Acid Alpha-Glucosidase Deficiency (Pompe Disease)

Approve for 1 year if the patient meets ALL of the following (A, B, C, D, E, and F):

ALL of the following

Age: Patient is ≥ 18 year of age>= 18 years
Weight: Patient weighs > 40 kg> 40 kg
Combination therapy: The medication will be used in combination with Pombiliti
Prior ERT non-improvement
- One of: Lumizyme (alglucosidase alfa) intravenous infusion
- One of: Nexviazyme (avalglucosidase alfa-ngpt) intravenous infusion
Diagnostic confirmation (ONE of)
- Laboratory test demonstrating deficient acid alpha-glucosidase activity in blood, fibroblasts, or muscle tissue
- Molecular genetic test demonstrating biallelic pathogenic or likely pathogenic acid alpha-glucosidase (GAA) gene variants
Prescriber specialty: Medication is prescribed by or in consultation with a geneticist, neurologist, a metabolic disorder sub-specialist, or a physician who specializes in the treatment of lysosomal storage disorders

Unapproved / Not Medically Necessary Uses

Conditions Not Covered

Opfolda is considered not medically necessary for ANY other use(s) including, but not limited to:

ANY of the following

Not covered examples: Gaucher Disease (an alternate dosage of miglustat is available for treatment of Gaucher disease)

Provider Actions & Prior Authorization

Prior Authorization

Prior authorization required

Prior authorization is recommended for prescription benefit coverage of Opfolda. All approvals are provided for 1 year.

Approval duration: 1 year

Documentation Required

Diagnostic documentation required

Provide laboratory evidence of deficient acid alpha-glucosidase activity OR a molecular genetic test demonstrating biallelic pathogenic or likely pathogenic acid alpha-glucosidase (GAA) gene variants to establish the diagnosis of late-onset Pompe disease.

Accepted specimen sources for enzyme activity testing: blood, fibroblasts, or muscle

Documentation Required

Prior therapy documentation

Document lack of improvement in objective measures after at least 1 year of prior enzyme replacement therapy with Lumizyme or Nexviazyme.

Example objective measures: forced vital capacity (FVC), six-minute walk test (6MWT)
Prior ERTs: Lumizyme (alglucosidase alfa), Nexviazyme (avalglucosidase alfa-ngpt)

Documentation Required

Prescriber specialty requirement

Opfolda must be prescribed by or in consultation with a geneticist, neurologist, a metabolic disorder sub-specialist, or a physician who specializes in the treatment of lysosomal storage disorders.

Clinical Evidence & Definitions

Prescribing information: Opfolda capsules, Amicus; September 2023.

Background note: Opfolda is described as an enzyme stabilizer for use in combination with Pombiliti and has no pharmacological activity as monotherapy for Pompe disease.

Coverage note: Opfolda is considered not medically necessary for other uses including Gaucher disease (an alternate miglustat dosage exists for that indication).

Objective measures: Examples include forced vital capacity (FVC) and six-minute walk test (6MWT).

Revision History

10/04/2023policy_created

New Policy created.

05/08/2024review

Early Annual Revision; genetic testing language rephrased to specify 'genetic test demonstrating biallelic pathogenic or likely pathogenic acid alpha-glucosidase gene variants'.

05/07/2025reviewLatest

Annual revision; No criteria changes.

Policy Summary

PayerCigna

PolicyOpfolda (miglustat) for Pompe disease

Policy CodePolicy N/A

Change TypeClarified; Annual revision

Effective Date

Next Review Date

Key ActionPrior authorization is recommended; approvals provided for 1 year.

On This Page

OpenPayer

Opfolda (miglustat) for Pompe disease

Coverage Summary

Unapproved / Not Medically Necessary Uses

Provider Actions & Prior Authorization

Applicable Codes

Clinical Evidence & Definitions

Key Thresholds

Revision History