Cipaglucosidase alfa-atga (Pombiliti) (Medicaid Expansion)

Policy ID: ME-I-9058-036-01

Section: Injections

Effective Date: March 01, 2025

Last Reviewed: January 14, 2025

Applies To: Medicaid Expansion Only

Description

Cipaglucosidase alfa-atga (Pombiliti ^TM ) are enzyme replacements used for specific indications as a treatment of Pompe disease. Pompe disease (glycogen storage disease type II, GSDII, glycogenosis type II, acid maltase deficiency) is an inherited disorder of glycogen metabolism caused by the absence or marked deficiency of acid alpha-glucosidase (GAA), an enzyme that degrades lysosomal glycogen.

Policy Application

All claims submitted for this policy will be processed according to the policy effective date and associated revision effective dates in effect on the date of service.

Criteria

Coverage is subject to the specific terms of the member's benefit plan.

Cipaglucosidase alfa-atga (Pombiliti) may be considered medically necessary when ALL the following criteria are met:

The individual must meet FDA-approved label for use (e.g., use outside of studied population will be considered investigational); and
Documentation of the individual's diagnosis must be submitted, as evidenced by the following:
- Deficiency of acid alpha-glucosidase enzyme activity (2% to 40% partial deficiency of GAA non-classic infantile forms or late onset forms) of the lab specific normal mean value; and
- Detection of pathogenic variants in the GAA gene by molecular genetic testing; and
The requested medication must be prescribed by, or in consult with, a cardiologist, neurologist or geneticist or specialist in Pompe disease; and
The individual must not have ventilation support for > 6 hours/day while awake; and
The individual has measurable signs of Pompe disease, such as impairment in pulmonary function or motor weakness; and
Documentation must be submitted of the individual's current motor function such as motor function, respiratory function as evidenced by scores from at least two of the following assessments:
- 6-minute walk test (6MWT); and
- Forced Vital Capacity (FVC) via Pulmonary Function Test; and
The individual must have failed a 6-month trial of Lumizyme and/or Nexviazyme, as evidenced by paid claims or pharmacy printouts; and
The individual has a 6MWT greater than or equal to 75 meters; and
The individual has a sitting FVC greater than or equal to 30% of the predicted value for healthy adults; and
Opfolda and Pombiliti must be taken concurrently

Reauthorization Criteria

The individual must have experienced and maintained clinical benefit since starting treatment with the requested medication, as evidenced by stabilization or improvement of the following:
- 6MWT and/or FVC

The use of cipaglucosidase alfa-atga (Pombiliti) for all other indications not listed in this policy is considered experimental/investigational and therefore non-covered because the safety and/or effectiveness cannot be established by the available published peer-reviewed literature.

Procedure codes

J1203

NOTE: In addition to the above criteria, product specific dosage and/or frequency limits may apply in accordance with the United States Food and Drug Administration (U.S. FDA)-approved product prescribing information, national compendia, Centers for Medicare and Medicaid Services (CMS) and other peer reviewed resources or evidence-based guidelines.

Diagnosis Codes

E74.02

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 01-14-2025 Effective March 01, 2025

Adopted Medicaid Expansion specific policy

Links

References (PDF)

Disclaimer

Current medical policy is to be used in determining a Member's contract benefits on the date that services are rendered. Contract language, including definitions and specific inclusions/exclusions, as well as state and federal law, must be considered in determining eligibility for coverage. Members must consult their applicable benefit plans or contact a Member Services representative for specific coverage information. Likewise, medical policy, which addresses the issue(s) in any specific case, should be considered before utilizing medical opinion in adjudication. Medical technology is constantly evolving, and the Company reserves the right to review and update medical policy periodically.

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