Alglucosidase alfa (Lumizyme) and Avalglucosidase alfa-ngpt (Nexviazyme) (Medicaid Expansion)

Policy ID: ME-I-58-023-03

Section: Injections

Effective Date: January 01, 2022

Revised Date: March 14, 2023

Revision Effective Date: April 01, 2023

Last Reviewed: March 23, 2023

Applies To: Medicaid Expansion Only

Description

Alglucosidase alfa (Lumizyme®) and avalglucosidase alfa-ngpt (Nexviazyme ^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 the lysosomal enzyme GAA.

In the late onset forms infantile-onset form, Pompe disease results in intralysosomal accumulation of glycogen in various tissues, particularly cardiac and skeletal muscles, and hepatic tissues, leading to the development of cardiomyopathy, progressive muscle weakness, and impairment of respiratory function.

In the juvenile and adult-onset forms, intralysosomal accumulation of glycogen is limited primarily to skeletal muscle, resulting in progressive muscle weakness.

Criteria

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

The use of alglucosidase alfa (Lumizyme) or avalglucosidase alfa-ngpt (Nexviazyme) may be considered medically necessary when ALL of 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 the area of Pompe disease; and
The individual must not have permanent invasive ventilation
Documentation must be submitted of the individual's current motor function such as motor function, respiratory function, cardiac involvement (infantile onset) and scores from at least two (2) of the following assessments:
- Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorder (CHOP-INTEND); or
- Hammersmith Infant Neurological Examination (HINE) Section 2 motor milestone score; or
- Hammersmith Functional Motor Scale Expanded (HFMSE); or
- Motor Function Measure - 32 items (MFM-32); or
- Revised Upper Limb Module (RULM); or
- 6-minute walk test (6MWT); or
- Forced Vital Capacity (FVC) via Pulmonary Function Test.

Initial Authorization: Six (6) months

Reauthorization Criteria

Continuation of therapy with alglucosidase alfa (Lumizyme) or avalglucosidase alfa-ngpt (Nexviazyme) may be considered medically necessary when ALL of the following are met:

The individual must have experienced and maintained clinical benefit since starting treatment with the requested medication, as evidenced by medical documentation (e.g., chart notes) attached to the request (subject to clinical review) including stabilization or improvement of the following:
- Motor function, respiratory function, cardiac involvement (infantile onset); or
- CHOP-INTEND, HINE, HFMSE, MFM-32, 6MWT, or RULM scores; or
- Forced Vital Capacity (FVC) via Pulmonary Function Test (ages 5 and older).

The use of alglucosidase alfa (Lumizyme) or avalglucosidase alfa-ngpt (Nexviazyme) 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

J0219

J0221

NOTE: In addition to the above criteria, product specific dosage and/or frequency limits may apply in accordance with the U.S. Food and Drug Administration (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 11-23-2021 Adopted Medicaid Expansion specific policy effective 1-1-2022

Internal Medical Policy Committee 3-23-2022 Added precertification drug avalglucosidase alfa-ngpt (Nexviazyme) to the policy effective April 1, 2022, and updated the title of the policy

Internal Medical Policy Committee 3-23-2023 Effective April 01, 2023

Updated criteria
Updated experimental/investigational statement

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.

Policy ID: ME-I-58-023-02

Section: Injections

Effective Date: January 01, 2022

Revised Date: March 23, 2022

Revision Effective Date: April 01, 2022

Last Reviewed: March 23, 2022

Archived Date: March 31, 2023

Applies To: Medicaid Expansion Only

Description

Alglucosidase alfa (Lumizyme®) and avalglucosidase alfa-ngpt (Nexviazyme^TM) are enzyme replacements used for specific indications as a treatment of Pompe disease.

In the juvenile and adult-onset forms, intralysosomal accumulation of glycogen is limited primarily to skeletal muscle, resulting in progressive muscle weakness.

Criteria

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

The use of alglucosidase alfa (Lumizyme) may be considered medically necessary when ALL of the following criteria are met:

The individual must meet criteria as outlined in prescribing information (PI) including recommendations for diagnosis and age; and
The prescriber is a, or in consult with, an endocrinologist, metabolic specialist or geneticist; and
The individual must have a diagnosis of Pompe disease confirmed by the following (as evidenced with submitted documentation):
- 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 individual must not have permanent invasive ventilation; and
The provider must submit documentation of the individual’s current motor function, as evidenced by scores from the following assessments:
- Infantile-onset disease:
  - muscle weakness, motor function, respiratory function, cardiac involvement, percent predicted forced vital capacity (FVC), and/or 6-minute walk test (6MWT); or
- Late-onset (non-infantile) disease:
  - 6-minute walk test (6MWT); and
  - Forced Vital Capacity (FVC) via Pulmonary Function Test.

Initial Authorization: 6 months

Reauthorization Criteria

Continuation of therapy with alglucosidase alfa (Lumizyme) may be considered medically necessary when ALL of the following are met:

The individual must not have permanent invasive ventilation; and
The individual must have experienced and maintained clinical benefit since starting treatment with the requested medication, as evidenced by medical documentation (e.g. chart notes) attached to the request (subject to clinical review) including improvement in the following scores and symptoms:
- Infantile-onset disease:
  - muscle weakness, motor function, respiratory function, cardiac involvement, percent predicted forced vital capacity (FVC), and/or 6-minute walk test (6MWT); or
- Late-onset (non-infantile) disease:
  - 6-minute walk test (6MWT); and
  - Forced Vital Capacity (FVC) via Pulmonary Function Test.

Continuation Authorization: 12 months

The use of alglucosidase alfa (Lumizyme) for any other indication is considered experimental/investigational because the safety and/or effectiveness of this service cannot be established by the available published peer-reviewed literature.

Procedure Codes

J0221

Avalglucosidase alfa-ngpt (Nexviazyme) may be considered medically necessary for individuals one (1) year of age and older with late-onset Pompe disease (GAA deficiency) when ALL of the following are met:

Diagnosis of late-onset Pompe disease based on:
- GAA enzyme assay which shows reduced enzyme activity (2% to 40% partial deficiency of the lab specific normal mean value); and
- Confirmed by ANY ONE of the following:
  - A second GAA enzyme activity assay in a separate sample (from purified lymphocytes or fibroblast [minimally invasive test]; or
  - A muscle biopsy, [ invasive testing]); or
  - GAA gene testing; and
- Presence of one or more clinical signs and symptoms of Pompe disease such as (but not limited to):
  - Progressive muscle weakness
  - Delayed motor function
  - Respiratory Insufficiency.

Reauthorization Criteria

Continuation of avalglucosidase alfa-ngpt (Nexviazyme) for the treatment of late-onset Pompe disease may be considered medically necessary when the initial criteria are met and the individual has documented positive clinical response (e.g. improvement in muscle function and mobility).

The use of avalglucosidase alfa-ngpt (Nexviazyme) for any other indication is considered experimental/investigational because the safety and/or effectiveness of this service cannot be established by the available published peer-reviewed literature.

Procedure Codes

J0219

Diagnosis Codes

E74.02

Professional Statements and Societal Positions Guidelines

Testing used to establish the diagnosis of Pompe disease

Acid alpha-glucosidase (GAA) enzyme activity. GAA enzyme activity analysis can be performed on dried blood spots thus permitting rapid and sensitive analysis. Standard conditions for assay of GAA in blood samples have been proposed by a Pompe Disease Diagnostic Working Group. Confirmation by measurement of GAA activity in another tissue (e.g., culture skin fibroblasts) or molecular analysis is recommended. Historically, measurement of GAA enzyme activity has been performed using cultured skin fibroblasts, but it may take four to six weeks to obtain results, and delayed diagnosis and initiation of treatment, particularly in infants, may affect outcome.

Complete deficiency (activity less than 1% of normal controls) of GAA enzyme activity is associated with classic infantile-onset Pompe disease.
Partial deficiency (activity 2% to 40% of normal controls) of GAA enzyme activity is associated with the non-classic infantile-onset and the late-onset

Note: (1) As a general rule, the lower the GAA enzyme activity the earlier the age of onset of disease. (2) GAA enzyme activity can be assayed in muscle; however, this invasive procedure usually requires anesthesia, which may not be tolerated in those who have infantile Pompe disease and cardiopulmonary compromise. (3) Peripheral leukocytes have been used to measure GAA enzyme activity but alternate isoenzymes such as maltase-glucoamylase may interfere with the assay. (4) GAA enzyme activity analysis can be performed on dried blood spots thus permitting rapid and sensitive analysis that is potentially useful for newborn screening

Acid alpha-glucosidase protein quantitation can be performed by an antibody-based method in cultured fibroblasts. Such testing may be important in determining if an affected individual produces cross-reactive immunologic material (CRIM). CRIM status affects response to ERT and CRIM-negative individuals need an altered plan for enzyme

Muscle biopsy. In contrast to the other glycogen storage disorders, GSD II is also a lysosomal storage disease. In GSD II glycogen storage may be observed in the lysosomes of muscle cells as vacuoles of varying severity that stain positively with periodic acid-Schiff. However, 20%-30% of individuals with late-onset Pompe disease with documented partial enzyme deficiency may not show these muscle-specific changes

Newborn screening. Newborn screening for GSD II, using GAA enzyme activity in dried blood spots as a primary screening tool.

ND Committee Review

Internal Medical Policy Committee 11-23-2021 Adopted Medicaid Expansion specific policy effective 1-1-2022

Internal Medical Policy Committee 3-23-2022 Added precertification drug avalglucosidase alfa-ngpt (Nexviazyme) to the policy effective April 1, 2022, and updated the title of the policy

Links

References (PDF)

Disclaimer

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Alglucosidase alfa (Lumizyme) and Avalglucosidase alfa-ngpt (Nexviazyme) (Medicaid Expansion)

Description

Criteria

Procedure Codes

Diagnosis Codes

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer

Description

Criteria

Procedure Codes

Procedure Codes

Diagnosis Codes

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer