Pombiliti (cipaglucosidase alfa-atga) for Pompe disease
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Cigna coverage policy governing prior authorization, medical necessity criteria, dosing, and coding for Pombiliti (cipaglucosidase alfa-atga) used with miglustat (Opfolda) to treat late-onset Pompe disease in adults >40 kg.
Genetic test language clarified to require 'biallelic pathogenic or likely pathogenic acid alpha-glucosidase (GAA) gene variants.'
The criterion regarding concomitant use with other medications used to treat Pompe disease was removed from Conditions Not Covered.
HCPCS code G0138 was added to coding.
Documentation requirements were added/updated to support criteria.
Coverage Criteria
FDA-Approved Indication (Initial Approval)
Approve for 1 year if the patient meets ALL of the following (A, B, C, D, E, and F):
Dosing: Each dose must not exceed 20 mg/kg administered IV no more frequently than once every 2 weeks.
Pombiliti (cipaglucosidase alfa-atga) for any use other than the FDA‑approved indication specified in this policy is not medically necessary. Requests for coverage that do not meet the clinical criteria and dosing requirements set forth in this policy will be denied as not covered.
Use of Pombiliti for indications other than the FDA‑approved combination indication — late‑onset Pompe disease in adults weighing > 40 kg treated in combination with Opfolda (miglustat) — is considered not medically necessary. The policy clarifies diagnostic confirmation requirements to specify a genetic test demonstrating biallelic pathogenic or likely pathogenic acid alpha‑glucosidase (GAA) gene variants. The prior policy exclusion that prohibited concomitant use with other Pompe disease medications has been removed.
Coding and Billing
Provider Actions and Requirements
Prior authorization required
Prior authorization is required for benefit coverage of Pombiliti; approval is recommended for patients who meet the Criteria and Dosing. Approvals are provided for the durations noted (initial approval: 1 year) and extended approvals are allowed if the patient continues to meet the Criteria and Dosing. Requests for doses outside the established dosing will be reviewed case-by-case by a clinician. Pombiliti must be prescribed by a physician who specializes in the condition being treated.
- Initial authorization duration: 1 year when criteria met
- Extended approvals allowed if patient continues to meet criteria and dosing
- Requests for nonstandard doses reviewed by clinician
Failure of prior enzyme replacement therapy required
Patient must have not demonstrated improvement in objective measures after receiving at least one year of either Lumizyme (alglucosidase alfa) or Nexviazyme (avalglucosidase alfangpt) prior to approval for Pombiliti.
- Objective measures examples include FVC and 6MWT
- Failure must be after ≥ 1 year of treatment with Lumizyme or Nexviazyme
Documentation required for diagnosis confirmation
Documentation is required where noted and may include chart notes, laboratory tests, claims records, prescription receipts, or other patient-specific information. Specifically, diagnosis confirmation requires either laboratory evidence of deficient acid alpha-glucosidase activity in blood, fibroblasts, or muscle tissue, or molecular genetic testing demonstrating biallelic pathogenic or likely pathogenic GAA gene variants.
- All documentation must include patient-specific identifying information
- Laboratory enzyme assay OR genetic test (biallelic pathogenic/likely pathogenic GAA variants) [documentation required]
Denial risk for missing covered codes
Requests for services not submitted with covered diagnosis or procedure codes will be denied as not covered.
- Submit requests with the applicable covered diagnosis and procedure (HCPCS/J) codes to avoid denial
Background
Pompe disease is a rare lysosomal storage disorder caused by deficiency of the enzyme acid alpha‑glucosidase (GAA), leading to accumulation of glycogen in muscle tissue. Clinical presentation varies with age of onset; late‑onset Pompe disease manifests any time after 12 months of age and typically features progressive skeletal muscle weakness and potential respiratory insufficiency. Diagnosis is established by either laboratory evidence of deficient GAA enzyme activity or genetic testing demonstrating biallelic pathogenic or likely pathogenic GAA variants. Pombiliti (cipaglucosidase alfa‑atga) is indicated in combination with miglustat (Opfolda) for adults > 40 kg with late‑onset Pompe disease who meet the prior‑therapy and diagnostic criteria described in this policy.
Definitions and Objective Measures
Initial Therapy Criteria
Initial therapy
Dose limit 20 mg/kg IV no more frequently than q2 weeks; approve for 1 year.
Continuation / Extension Criteria
Continuation/Extension
Extended approval
Approvals provided for the duration noted (initial 1 year). Documentation as required in initial criteria must be maintained; prescriber must be a specialist or consult one.
Step Therapy Requirements
| Required prior ERT trial | Minimum duration | Failure definition |
|---|---|---|
| Lumizyme (alglucosidase alfa) | At least 1 year | Patient has not demonstrated an improvement in objective measures (e.g., FVC or 6MWT) after treatment |
| Nexviazyme (avalglucosidase alfangpt) | At least 1 year | Patient has not demonstrated an improvement in objective measures (e.g., FVC or 6MWT) after treatment |
Quantity Limits
Site of Care and Administration
Infusion center coding (G0138)
Administration is described as an intravenous infusion; coding includes HCPCS G0138 which covers IV infusion of cipaglucosidase alfa-atga and includes provider/supplier acquisition and clinical supervision of oral miglustat administration.
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