CurrentMass General Brigham Health PlanPolicy N/A

Pombiliti (cipaglucosidase alfa-atga) coverage for late-onset Pompe disease

Medical-benefit prior authorization policy for Pombiliti used with Opfolda for adults with late-onset Pompe disease; applies to MassHealth members and governs prescriber requirements, diagnostic confirmation, prior treatment failure, dosing, and authorization duration.

Policy Summary

PayerMass General Brigham Health Plan

PolicyPombiliti (cipaglucosidase alfa-atga) coverage for late-onset Pompe disease

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionSubmit prior authorization through the medical benefit with specialist documentation, diagnostic confirmation of GAA deficiency, current weight, and evidence of prior inadequate response or contraindication to Lumizyme or Nexviazyme.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

≥18minimum patient age

≥40 kgminimum patient weight

1 yearapproval duration

Medicalbenefit type

Requires Opfolda

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Coverage Criteria for Pombiliti (with Opfolda)

Initial Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met

Primary eligibility: Member has diagnosis of late-onset Pompe Disease

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

Weight: Member's current weight is >= 40 kg>= 40 kg

Diagnostic confirmation: One of: results from GAA assay on cultured skin fibroblasts showing reduced or absent activity; lymphocyte testing; blood spot assay; or genetic testing confirming mutation in GAA gene

Age: Member is >= 18 years of age>= 18 years

Prior therapy requirement: Inadequate response or adverse reaction to ONE, or contraindication to BOTH: Lumizyme; Nexviazyme

Lumizyme or Nexviazyme should not be used concurrently with Opfolda and Pombiliti

Combination therapy: Requested agent will be used in combination with Opfolda (miglustat 65 mg)

Dosing is appropriate within the FDA labeling

Initial therapy requirements — 3 top-level nodes

Indication: Indicated in combination with Opfolda for adult patients with late-onset Pompe disease weighing >= 40 kg who are not improving on current ERT

Prior ERT failure: Inadequate response or adverse reaction to ONE, or contraindication to BOTH Lumizyme and Nexviazyme

Diagnostic confirmation: Confirm with GAA assay, lymphocyte testing, blood spot assay, or genetic testing

Continuation Therapy — Covered for continuation when prescriber resubmits and infers positive response

Covered for continuation when prescriber resubmits and infers positive response

Resubmission by prescriber will infer a positive response to therapy

Approvals may be granted for 1 year

Continuation — Continuation approvals

Positive response: Resubmission by prescriber will infer a positive response to therapy

Approvals may be granted for 1 year

Lumizyme (alglucosidase alfa) and Nexviazyme (avalglucosidase alfa) should not be administered concurrently with the combination of Pombiliti and Opfolda. The policy explicitly states that concurrent use of Lumizyme or Nexviazyme with Opfolda and Pombiliti is not permitted, and prior authorization for Pombiliti in combination with Opfolda assumes that members meet the policy’s prior therapy requirements (inadequate response/adverse reaction to one, or contraindication to both, Lumizyme and Nexviazyme) rather than concurrent treatment.

Patient Eligibility and Coding Parameters

inv-04: Weight — >= 40 kg

Weight thresholdMember's current weight is >= 40 kg

Source contextPombiliti is indicated for adult patients with late-onset Pompe disease weighing >=40 kg

Related clinical requirementWeight must be documented in prior authorization submission

inv-05: Age — >= 18 years

Age thresholdMember is >= 18 years of age

PopulationPolicy applies to adult patients with late-onset Pompe disease

Documentation

Prescriber and Documentation Requirements

Prior Authorization

Prior authorization required

Prior authorization is required through the medical benefit for Pombiliti when used in combination with Opfolda for eligible adults with late-onset Pompe disease.

Step Therapy

Pre-treatment requirement (prior ERT failure)

Not a formal step therapy policy, but coverage requires inadequate response or adverse reaction to ONE, or contraindication to BOTH, Lumizyme and Nexviazyme before approval of Pombiliti.

Inadequate response or adverse reaction to ONE: Lumizyme or Nexviazyme
Or contraindication to BOTH: Lumizyme and Nexviazyme

Key Definitions

inv-11: Late-onset Pompe disease — definition and diagnostic confirmation (GAA assay, lymphocyte testing, etc.)

DefinitionLate-onset Pompe disease is a lysosomal acid alpha-glucosidase (GAA) deficiency causing progressive muscle weakness; policy applies to adult patients not improving on current ERT

Diagnostic confirmation — accepted testsOne of: GAA assay on cultured skin fibroblasts showing reduced/absent activity; lymphocyte testing; blood spot assay; or genetic testing confirming a GAA mutation

Prescriber requirementPrescriber is a specialist in genetic or metabolic diseases or specialist consult notes must be provided

Treatment contextPombiliti must be used in combination with Opfolda (miglustat 65 mg) for this indication

Background and Clinical Context

Late-onset Pompe disease is a lysosomal acid alpha-glucosidase (GAA) deficiency that leads to progressive muscle weakness and is treated with enzyme replacement therapy. Pombiliti (cipaglucosidase alfa-atga) is indicated for adult patients with late-onset Pompe disease who meet the policy thresholds — specifically, patients must be ≥ 18 years of age and weigh ≥ 40 kg. Pombiliti must be used in combination with Opfolda (miglustat 65 mg), and coverage is intended for patients who are not improving on their current ERT, with documentation of inadequate response or adverse reaction to one, or contraindication to both, Lumizyme and Nexviazyme.

Place of Service and Billing

Billing Rule

Infusion center — medical benefit prior authorization

Pombiliti is covered under the medical benefit only; prior authorization requests must be submitted via the medical benefit contact phone or fax provided.

Medical benefit prior authorization phone: 833-895-2611
Medical benefit prior authorization fax: 888-656-6671