Vestronidase alfa-vjbk (Mepsevii) (Medicaid Expansion)

Policy ID: ME-I-188-005-04

Section: Injections

Effective Date: January 01, 2022

Revised Date: March 09, 2025

Revision Effective Date: May 01, 2025

Last Reviewed: March 11, 2025

Applies To: Medicaid Expansion Only

Description

Vestronidase alfa-vjbk (Mepsevii) is a recombinant human lysosomal beta glucuronidase enzyme replacement therapy indicated in pediatric and adult individuals for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome).

MPS VII is caused by pathogenic mutations in the GUSB gene. The GUSB gene is responsible for producing an enzyme called beta-glucuronidase which is involved in the breakdown of large molecules called glycosaminoglycans (GAGs).

Policy Application

All claims submitted under this policy's section 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.

The use of vestronidase alfa-vjbk (Mepsevii) 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
Vestronidase alfa-vjbk (Mepsevii) must be prescribed by, or in consult with, an expert in lysosomal storage diseases; and
The member must not have had a hematopoietic stem cell transplant ; and
If 3 years of age or older, must be able to walk without support; and
One or more of the following must be submitted:
- Elevated level of urinary excretion of glycosaminoglycans (GAGs) such as chondroitin sulfate and dermatan sulfate, as defined by being 2 or more times normal; or
- Hepatomegaly (liver size 1.25 or more times normal); or
- Splenomegaly (spleen size 5 or more times normal); 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 vestronidase alfa-vjbk (Mepsevii) may be considered medically necessary when the following is met:

The individual must have experienced meaningful clinical benefit since starting treatment with vestronidase alfa-vjbk (Mepsevii), as evidenced by medical documentation (e.g., chart notes) attached to the request (subject to clinical review) including improvement in the one of the following scores and symptoms:
- Stability or improvement in Skeletal abnormalities shown on x-ray, short stature, macrocephaly; or
- Urinary glycosaminoglycan (uGAG) levels normalization defined by laboratory reference range ; or
- Reduction in liver volume to normal size or by 10%; or
- Reduction in spleen volume by 15%; or
- Stability or improvement in six (6)-minute walk test (6MWT); or
- Stability or improvement in Forced Vital Capacity (FVC) via Pulmonary Function Test.

The use of vestronidase alfa-vjbk (Mepsevii) 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 Code

J3397

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 Code

E76.29

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 11-23-2021 - Effective January 01, 2022

Adopted Medicaid Expansion specific policy

Internal Medical Policy Committee 11-29-2022 - Effective January 01, 2023

Updated initial criteria, and
Updated experimental/investigational statement

Internal Medical Policy Committee 11-15-2023 Annual Review-no changes in criteria

Internal Medical Policy Committee 11-19-2024 - Effective January 01, 2025

Annual review no clinical content change; and
Added Policy Application

Internal Medical Policy Committee 03-11-2025 Effective May 01, 2025

Updated initial and reauthorization criteria required based on the DHHS PDL Version 2025.3

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-188-005-03

Section: Injections

Effective Date: January 01, 2022

Revised Date: October 18, 2024

Revision Effective Date: January 01, 2025

Last Reviewed: November 19, 2024

Archived Date: April 30, 2025

Applies To: Medicaid Expansion Only

Description

Policy Application

All claims submitted under this policy's section 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.

The use of vestronidase alfa-vjbk (Mepsevii) 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
Vestronidase alfa-vjbk (Mepsevii) must be prescribed by, or in consult with, an expert in lysosomal storage diseases; and
Documentation of the member's diagnosis must be submitted, as evidenced by the following:
- Deficiency of beta-glucuronidase enzyme
- Detection of pathogenic variants in the GUSB gene by molecular genetic testing; and
The provider must submit documentation of one or more of the following:
- Skeletal abnormalities
- Elevated level of urinary excretion of glycosaminoglycans (GAGs) such as chondroitin sulfate and dermatan sulfate, as defined by being above the upper limit of normal by the laboratory reference range
- Liver and/or spleen volume
- Six (6)-minute walk test (6MWT)
- Motor function test (e.g., Bruininks-Oseretsky Test of Motor Proficiency (BOT-2))
- Forced Vital Capacity (FVC) via Pulmonary Function Test.

Initial Authorization: Six (6) months

Reauthorization Criteria

Continuation of therapy with vestronidase alfa-vjbk (Mepsevii) may be considered medically necessary when the following is met:

The individual must have experienced meaningful clinical benefit since starting treatment with vestronidase alfa-vjbk (Mepsevii), as evidenced by medical documentation (e.g., chart notes) attached to the request (subject to clinical review) including improvement in the one of the following scores and symptoms:
- Stability or improvement in Skeletal abnormalities shown on x-ray, short stature, macrocephaly; or
- Reduction in urinary excretion of glycosaminoglycans (GAGs); or
- Reduction in liver and/or spleen volume; or
- Stability or improvement in six (6)-minute walk test (6MWT); or
- Stability or improvement in Forced Vital Capacity (FVC) via Pulmonary Function Test.

Procedure Code

J3397

Diagnosis Code

E76.29

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 11-23-2021 - Effective January 01, 2022

Adopted Medicaid Expansion specific policy

Internal Medical Policy Committee 11-29-2022 - Effective January 01, 2023

Updated initial criteria, and
Updated experimental/investigational statement

Internal Medical Policy Committee 11-15-2023 Annual Review-no changes in criteria

Internal Medical Policy Committee 11-19-2024 - Effective January 01, 2025

Annual review no clinical content change; and
Added Policy Application

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Vestronidase alfa-vjbk (Mepsevii) (Medicaid Expansion)

Description

Policy Application

Criteria

Procedure Code

Diagnosis Code

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer

Description

Policy Application

Criteria

Procedure Code

Diagnosis Code

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer