CurrentCareSourcePolicy N/A

UTILIZATION MANAGEMENT MEDICAL POLICY

Defines medical-benefit coverage criteria, dosing limits, prior authorization and exclusions for Naglazyme (galsulfase) for treatment of Mucopolysaccharidosis type VI (MPS VI / Maroteaux-Lamy syndrome). Applies to medical infusion coverage and specifies required diagnostic confirmation and prescriber specialty.

Policy Summary

PayerCareSource

PolicyUTILIZATION MANAGEMENT MEDICAL POLICY

Policy CodePolicy N/A

Change Typeclarified

Effective Date

Next Review Date

Key ActionPrior authorization is recommended for medical benefit coverage; approve for 1 year if criteria met.

SourceLink

POLICY UPDATE CHANGES

Genetic confirmation language previously revised to specifically state biallelic pathogenic or likely pathogenic ARSB gene variants (historical note).

1Covered Indications (MPS VI)

1 mg/kgMax dose per administration

weeklyMax dosing frequency

Coverage Summary

Defines medical-benefit coverage criteria, dosing limits, prior authorization, diagnostic confirmation, prescriber specialty requirements, and exclusions for Naglazyme (galsulfase) for treatment of Mucopolysaccharidosis type VI (MPS VI / Maroteaux-Lamy syndrome) when administered as a medical intravenous infusion; the product is covered with criteria and subject to prior authorization and dosing limits.

FDA-Approved Indication — Authorization Criteria

FDA-Approved Indication - Authorization Criteria

Approve for 1 year if the patient meets BOTH of the following (A and B):

ALL of the following

A) Diagnosis established by ONE of the following:
- i.: Laboratory test demonstrating deficient N-acetylgalactosamine 4-sulfatase (arylsulfatase B) activity in leukocytes or fibroblasts.
- ii.: Molecular genetic test demonstrating biallelic pathogenic or likely pathogenic arylsulfatase B (ARSB) gene variants.
B): Naglazyme is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder sub-specialist, or physician who specializes in the treatment of lysosomal storage disorders.
Dosing: Each dose must not exceed 1 mg/kg administered intravenously no more frequently than once weekly.<= 1 mg/kg per dose, IV no more frequently than once weekly
Approvals are provided for the duration noted (1 year). Requests for doses outside established dosing will be considered case-by-case by a clinician.

Conditions Not Recommended for Approval

Coverage is not recommended when the following apply:

ANY of the following

Coverage is not recommended for circumstances not listed in the Recommended Authorization Criteria.
Criteria will be updated as new published data are available.

Provider Actions

Prior Authorization

Prior authorization required

Prior authorization is recommended for medical benefit coverage; approve for 1 year if criteria met. Requests for doses outside the dosing documented will be considered case-by-case by a clinician (Medical Director or Pharmacist).

Approve for 1 year when criteria met

Documentation Required

Diagnostic documentation required

Provider must document either deficient arylsulfatase B enzyme activity in leukocytes or fibroblasts OR molecular genetic test demonstrating biallelic pathogenic or likely pathogenic ARSB variants, and that prescriber is an appropriate specialist or consulted one.

Deficient arylsulfatase B enzyme activity in leukocytes or fibroblasts
OR molecular genetic test showing biallelic pathogenic or likely pathogenic ARSB variants
Prescriber is appropriate specialist or consulted one.

Billing Rule

Approval duration

Approvals are provided for the duration noted (policy indicates 1 year approval when criteria met).

Dosing outside documented dosing will be reviewed case-by-case by a clinician.

Background

Naglazyme (galsulfase) is recombinant human N-acetylgalactosamine 4-sulfatase indicated for Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome). The definitive diagnosis is established by either demonstrating deficient arylsulfatase B (N-acetylgalactosamine 4-sulfatase) activity in leukocytes or fibroblasts or by genetic testing demonstrating pathogenic ARSB variants. Enzyme replacement therapy with Naglazyme has been shown to improve walking and stair-climbing capacity; hematopoietic stem cell transplant is an alternative definitive treatment in select patients who are intolerant of or do not respond to enzyme replacement therapy.

Biallelic pathogenic or likely pathogenic ARSB gene variants: Presence of pathogenic or likely pathogenic variants in both alleles of the ARSB gene demonstrated by molecular genetic testing.

Clinical Evidence

No formal evidence items were provided in the brief (evidence list is empty). The clinical-effectiveness statement in the background notes that enzyme replacement with Naglazyme improves walking and stair-climbing capacity; hematopoietic stem cell transplantation is an alternative in select cases (typically reserved for those intolerant of or unresponsive to enzyme replacement).

Revision History

04/12/2023Mucopolysaccharidosis Type VI (Maroteaux - Lamy Syndrome): Confirmation of genetic mutation in the arylsulfatase B gene was revised to more specifically state, genetic confirmation of biallelic pathogenic or likely pathogenic arylsulfatase gene variants testing.

Genetic confirmation language revised to specifically state biallelic pathogenic or likely pathogenic ARSB gene variants (historical note).

04/24/2024Annual Revision, Summary of Changes = No criteria changes.

History note indicating no criteria changes in 2024.

04/16/2025Annual Revision, Summary of Changes = No criteria changes.Latest

2025 annual review: no criteria changes.