CurrentMass General Brigham Health PlanPolicy N/A

Pombiliti (cipaglucosidase alfa-atga) — coverage criteria

Defines prior authorization, coverage criteria, and limitations for Pombiliti (cipaglucosidase alfa-atga) used with Opfolda in adults with late-onset Pompe disease within Mass General Brigham Health Plan.

Policy Summary

PayerMass General Brigham Health Plan

PolicyPombiliti (cipaglucosidase alfa-atga) — coverage criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization through the medical benefit; approvals may be granted for up to 1 year when all coverage criteria are met.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

prior authauthorization requirement

1 yeartypical approval duration

with Opfoldacombination therapy

adultsage group

≥240 kgweight requirement

Coverage Criteria for Pombiliti (cipaglucosidase alfa-atga)

Initial Authorization Criteria

Covered when ALL of the following are met:

Primary criteria: Member has a diagnosis of late-onset Pompe disease

Age requirement: Member is an adult (age referenced in policy)

Weight requirement: Member's current weight is >= 240 kg>= 240 kg

Prescriber requirement: Prescriber is a specialist in genetic or metabolic diseases or specialist consult notes are provided

Policy Summary

PayerMass General Brigham Health Plan

PolicyPombiliti (cipaglucosidase alfa-atga) — coverage criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization through the medical benefit; approvals may be granted for up to 1 year when all coverage criteria are met.

SourceLink

On This Page

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Provider Actions, Authorization & Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required for Pombiliti (cipaglucosidase alfa‑atga). Typical approvals may be granted for up to 1 year; prior authorization must be obtained before initiating therapy.

Prior authorization required
Typical approval duration: up to 1 year

Step Therapy

Step / Prior Therapy Requirement

Step therapy / prior enzyme replacement therapy (ERT) requirement applies. Approval generally requires inadequate response or adverse reaction to ONE, or contraindication to BOTH listed ERTs.

Step requirement: trial of Lumizyme (alglucosidase alfa) and/or Nexviazyme (avalglucosidase alfa) as applicable
Exception: member with inadequate response or adverse reaction to ONE, or contraindication to BOTH Lumizyme and Nexviazyme

Documentation Required

Required Documentation

Clinical documentation must be submitted to support medical necessity. Include prescriber and diagnostic details to facilitate review.

Specialist prescriber information (geneticist or metabolic disease specialist) or consult notes from such specialist
Clinical consult notes outlining disease history and rationale for Pombiliti
Diagnostic confirmation of late‑onset Pompe disease: one of the following - reduced/absent GAA activity from cultured skin fibroblasts, lymphocyte testing, dried blood spot assay, or GAA gene mutation on genetic testing
Current weight (member ≥ 40 kg) and age documentation (member ≥ 18 years)
Documentation of prior ERT trials, inadequate response or adverse reaction, or contraindication to Lumizyme and/or Nexviazyme when applicable
Planned dosing consistent with FDA labeling and statement whether therapy will be used with miglustat (Opfolda)

Denial Risk

Criteria Non‑fulfillment / Denial Risk

Requests may be denied if required criteria are not met. Ensure all of the items below are documented and supported in the medical record prior to submission.

Diagnosis not documented as late‑onset Pompe disease
Prescriber is not a metabolic/genetic specialist and no specialist consult notes provided
Member weight < 40 kg or weight not documented
No diagnostic confirmation via GAA assay, lymphocyte testing, blood spot assay, or genetic testing
Member age < 18 years or age not documented
No documentation of prior ERT trial, inadequate response/adverse reaction, or documented contraindication to Lumizyme and/or Nexviazyme when step requirement applies
Planned dosing inconsistent with FDA labeling or insufficient dosing details
Concurrent use of Lumizyme or Nexviazyme with Opfolda (miglustat) when prohibited

Background

Late-onset Pompe disease is a lysosomal acid alpha-glucosidase (GAA) deficiency characterized by progressive muscle weakness. This policy addresses treatment with Pombiliti (cipaglucosidase alfa-atga), which per the coverage guidelines must be used in combination with Opfolda (miglustat 65 mg) for adult patients. Authorization is contingent on confirming the diagnosis (e.g., GAA assay from cultured skin fibroblasts, lymphocyte testing, blood spot assay, or genetic testing) and meeting other criteria such as specialist prescriber involvement, weight and age requirements, appropriate dosing within FDA labeling, and prior inadequate response or contraindication to alternative ERTs.

Definitions

Late‑onset Pompe Disease

DefinitionLate‑onset Pompe disease is a lysosomal acid alpha‑glucosidase (GAA) deficiency causing progressive muscle weakness

Diagnostic confirmation — primary methodGAA assay from cultured skin fibroblasts showing reduced or absent activity

Diagnostic confirmation — alternative methodsLymphocyte testing, blood spot assay, or genetic testing confirming a GAA mutation

Opfolda

DefinitionOpfolda (miglustat 65 mg) — oral therapy required to be used in combination with Pombiliti per this policy

Combination requirementRequested Pombiliti must be used in combination with Opfolda (miglustat 65 mg) as a condition of authorization

Concurrent therapy noteLumizyme or Nexviazyme should not be used concurrently with Opfolda and Pombiliti

OpenPayer

Pombiliti (cipaglucosidase alfa-atga) — coverage criteria

Coverage Criteria for Pombiliti (cipaglucosidase alfa-atga)

Coding, Age, and Weight Requirements

Provider Actions, Authorization & Documentation

Background

Definitions