ModifiedColorado Rocky Mountain Health PlansPolicy 2025 P 1420-3

Opfolda® (miglustat) - Prior Authorization/Notification - UnitedHealthcare Commercial Plans

Defines prior authorization and reauthorization clinical criteria for Opfolda (miglustat) when used in combination with Pombiliti for treatment of adult patients with late-onset Pompe disease under UnitedHealthcare commercial plans.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyOpfolda® (miglustat) - Prior Authorization/Notification - UnitedHealthcare Commercial Plans

Policy CodePolicy 2025 P 1420-3

Change TypeAnnual review (no coverage change)

Effective DateFeb 15, 2026

Next Review Date

Key ActionObtain prior authorization documenting diagnosis of late-onset Pompe disease and an active UnitedHealthcare medical benefit prior authorization for Pombiliti; approvals issued for 12 months.

SourceLink

POLICY UPDATE CHANGES

New program created 11/2023.

Criteria clarified without change to clinical intent 11/2024.

Annual review 11/2025 with no change to coverage criteria.

2Required approval criteria

12Authorization duration (months)

>=40Minimum patient weight (kg)

Coverage Summary

Scope: This policy defines prior authorization and reauthorization clinical criteria for Opfolda (miglustat) when used in combination with Pombiliti for treatment of adult patients with late-onset Pompe disease under UnitedHealthcare commercial plans. Coverage stance: covered_with_criteria.

Background summary: Opfolda (miglustat) is an enzyme stabilizer indicated, in combination with Pombiliti, for treatment of adult patients with late-onset Pompe disease (GAA deficiency) weighing ≥40 kg who are not improving on their current enzyme replacement therapy. Initial authorization requires diagnosis of late-onset Pompe disease and an active UnitedHealthcare medical benefit prior authorization for Pombiliti; reauthorization requires documentation of positive clinical response to Opfolda plus Pombiliti and continued concomitant use. Authorizations are issued for 12 months.

Reauthorization Criteria

Reauthorization

Opfolda will be approved based on both of the following criteria:

Documentation of positive clinical response to Opfolda plus Pombiliti
Opfolda continues to be prescribed in combination with Pombiliti

Provider Actions & Requirements

Prior Authorization

Prior authorization required

Initial approval requires documentation of diagnosis of late-onset Pompe disease and an active UnitedHealthcare medical benefit prior authorization for Pombiliti; authorization issued for 12 months.

Diagnosis: late-onset Pompe disease
Active UnitedHealthcare medical benefit prior authorization for Pombiliti

Prior Authorization

Reauthorization requirements

Reauthorization requires documentation of positive clinical response to Opfolda plus Pombiliti and continued prescription in combination with Pombiliti; authorization issued for 12 months.

Documentation Required

Automated approval note

UnitedHealthcare may approve initial and re-authorization based solely on previous claim/medication history, diagnosis codes (ICD-10) and/or claim logic; automated processes vary by program and/or therapeutic class.

Operational note: Pombiliti (cipaglucosidase alfa-atga) must be an active ERT prior authorization/medical benefit and is required as concomitant therapy per this policy. Initial approval requires an active UnitedHealthcare medical benefit prior authorization for Pombiliti for treatment of late-onset Pompe disease.

See definitions entry for the term Pombiliti for the formal definition.

Background & Definitions

Opfolda (miglustat) is an enzyme stabilizer indicated in combination with Pombiliti for treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg who are not improving on their current enzyme replacement therapy (ERT).

Pombiliti: cipaglucosidase alfa-atga, a hydrolytic lysosomal glycogen-specific enzyme used as enzyme replacement therapy (ERT) for late-onset Pompe disease.

Revision History

11/2023program_created