CurrentCommunity-CarePolicy N/A

Cipaglucosidase alfa (Pombiliti) for late-onset Pompe disease — Coverage Criteria

Defines medical benefit coverage criteria for initiation and continuation of cipaglucosidase alfa (Pombiliti) for members with late-onset Pompe disease, and applies to providers seeking authorization under the payer's medical benefit medication policy.

Policy Summary

PayerCommunity-Care

PolicyCipaglucosidase alfa (Pombiliti) for late-onset Pompe disease — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization demonstrating diagnosis confirmation, specialist prescriber, miglustat co-prescription, and symptomatic disease.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1 yearauthorization period if approved

Nonequantity limits

J1202HCPCS code

Mar 2024last reviewed

Coverage Criteria for Cipaglucosidase alfa (Pombiliti)

Initial Therapy

Covered when ALL of the following are met for initial approval:

Initial Approval - Late-onset Pompe disease: Clinical diagnosis of late-onset Pompe disease confirmed by genetic sequencing OR acid alpha-glucosidase enzyme assay.

Weight is required for approval.

Prescriber Requirement: Prescribed by a medical geneticist or neuromuscular specialist.

Concomitant Therapy: Miglustat is prescribed with cipaglucosidase alfa (Pombiliti).

Clinical Status: Patient is experiencing symptoms or objective signs of Pompe disease.

Policy Summary

PayerCommunity-Care

PolicyCipaglucosidase alfa (Pombiliti) for late-onset Pompe disease — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization demonstrating diagnosis confirmation, specialist prescriber, miglustat co-prescription, and symptomatic disease.

SourceLink

On This Page

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Continuation Therapy

Covered when ALL of the following are met for continuation of therapy:

Ongoing Diagnosis: Ongoing diagnosis of late-onset Pompe disease.

Clinical Benefit: Member is benefiting from therapy, is being monitored, and is appropriate to continue therapy.

Authorization period up to one year if approved.

This policy defines coverage criteria for cipaglucosidase alfa (Pombiliti) in members with late-onset Pompe disease. For initial approval, coverage requires confirmation of the clinical diagnosis by genetic sequencing OR acid alpha-glucosidase enzyme assay, prescription by a medical geneticist or neuromuscular specialist, co-prescription of miglustat with cipaglucosidase alfa, and that the patient is experiencing symptoms or objective signs of Pompe disease. Documentation of weight is also required for approval.

For continuation of therapy, coverage is allowed when the member has an ongoing diagnosis of late-onset Pompe disease and is benefiting from therapy, is being monitored, and is clinically appropriate to continue treatment. If approved, authorization is allowed for up to one year. There are no quantity limits specified for treatment of late-onset Pompe disease.

Confirmation of late-onset Pompe disease

Diagnostic genetic testing or biochemical assay required to confirm diagnosis prior to coverage of therapy:

Confirmation of late-onset Pompe disease: Diagnostic confirmation by ONE of the following: genetic sequencing OR acid alpha-glucosidase enzyme assay.

Required for coverage of therapy.

Coding and Billing

Applicable HCPCS CodesHCPCS

J1202

HCPCS Code listed (HCPCS Code Description not provided in document)

Weight

RequirementA weight measurement is required for approval.

ContextListed under Quantity Limits for late-onset Pompe disease in the policy.

UseProvide current patient weight with prior authorization request to support dosing and coverage decision.

Provider Actions and Authorization Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Requests must demonstrate that initial approval criteria are met for late-onset Pompe disease before services or medication are provided.

Prior authorization required for initial and continuation therapy
Authorization period: up to 1 year if approved
Weight required for approval

Documentation Required

Required Documentation

Clinical documentation must be submitted with the prior authorization request to support the diagnosis and medical necessity.

Confirmation of diagnosis via genetic sequencing OR acid alpha-glucosidase enzyme assay (lab report or genetic test result)
Prescriber specialty documented: medical geneticist or neuromuscular specialist
Documentation of symptoms or objective signs of Pompe disease and clinical rationale for therapy
For continuation requests, documentation the member is benefiting from therapy and is being monitored
Weight (current) for the patient

Denial Risk

Triggers for Denial

Requests may be denied if required diagnostic confirmation, specialist prescriber, or other required documentation is not provided.

No documented genetic sequencing or acid alpha-glucosidase enzyme assay confirming late-onset Pompe disease
Prescriber is not a medical geneticist or neuromuscular specialist
Lack of documentation showing the patient is symptomatic or demonstrating objective signs of Pompe disease
For continuation, absence of documentation showing clinical benefit or monitoring

Note

Provider Action Checklist

Provider actions — ensure criteria and documentation are submitted with the authorization request and consult payer requirements for any additional form(s) or supporting records.

Submit prior authorization request with required documentation listed above
Ensure prescriber specialty is clearly indicated (medical geneticist or neuromuscular specialist)
Include recent patient weight and clinical monitoring notes for continuation requests
Confirm miglustat is prescribed with cipaglucosidase alfa (Pombiliti) when applicable

Covered Indications

Confirmation of late-onset Pompe disease

Diagnostic genetic testing or biochemical assay required to confirm diagnosis prior to coverage of therapy:

Confirmation of late-onset Pompe disease: Diagnostic confirmation by ONE of the following: genetic sequencing OR acid alpha-glucosidase enzyme assay.

Required for coverage of therapy.

Clinical Definitions

Clinical diagnosis

DefinitionClinical diagnosis of late-onset Pompe disease must be confirmed by genetic sequencing OR acid alpha-glucosidase enzyme assay.

Prescriber requirementMust be prescribed by a medical geneticist or neuromuscular specialist.

Treatment requirementMiglustat must be prescribed with cipaglucosidase alfa (Pombiliti).

Clinical statusPatient must be experiencing symptoms or objective signs of Pompe disease at initiation.

Eligibility Requirements

There are no additional top-level eligibility requirement nodes specified in this policy beyond the coverage criteria for initial and continuation therapy. Eligibility for coverage is determined by meeting the listed diagnostic, prescriber, concomitant medication, and clinical benefit requirements.

Background

Late-onset Pompe disease is a lysosomal storage disorder for which diagnosis can be established by either genetic sequencing or by measurement of acid alpha-glucosidase enzyme activity. This policy requires a confirmed diagnosis by one of these methods and that treatment with cipaglucosidase alfa be prescribed by a medical geneticist or neuromuscular specialist. The policy also requires that miglustat be prescribed concomitantly and that the patient have symptoms or objective findings consistent with Pompe disease before initiating therapy.