Publish Date:

10/23/2023

Last Review Date:

09/11/2023

Overview

• Coding
• References
• Clinical criteria
• Document history

Overview

This document addresses Naglazyme (galsulfase), an enzyme replacement therapy approved by the Food and Drug Administration (FDA) to treat individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

The mucopolysaccharidoses are a group of inherited metabolic diseases caused by the deficiency of lysosomal enzymes needed to breakdown mucopolysaccharides or glycosaminoglycan (GAGs). The progressive accumulation of GAGs in lysosomes leads to respiratory, cardiac, skeletal and connectivity, neurologic and ophthalmologic complications. There are seven distinct types of mucopolysaccharidosis (I, II, III, IV, VI, VII, and IX). Accurate diagnosis is important to provide disease-specific enzyme replacement therapy. Diagnosis is confirmed through urinary GAG concentration measurement, enzymatic activity measurement or genetic testing.

Clinical Criteria

When a drug is being reviewed for coverage under a member’s medical benefit plan or is otherwise subject to clinical review (including prior authorization), the following criteria will be used to determine whether the drug meets any applicable medical necessity requirements for the intended/prescribed purpose.

Naglazyme (galsulfase)

Initial requests for Naglazyme (galsulfase) may be approved if the following criteria are met:

1. Individual has a diagnosis of Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) demonstrated by (Akyol 2019, Wood 2012):
   • Documentation is provided showing an increase in dermatan sulfate in the urine and a decrease in the activity of N-acetylgalactosamine-4-sulfatase (arylsulfatase B) enzyme as measured in fibroblasts or leukocytes combined with normal enzyme activity level of another sulfatase; OR
   • Documentation is provided showing an N-acetylgalactosamine-4-sulfatase (arylsulfatase B) gene mutation.

Continuation requests for Naglazyme (galsulfase) may be approved if the following criterion is met:

1. Documentation is provided that there is clinically significant improvement or stabilization in clinical signs and symptoms of disease (including but not limited to reduction in urinary GAG excretion, reduction in hepatosplenomegaly, improvement in pulmonary function, improvement in walking distance and/or improvement in fine or gross motor function) compared to the predicted natural history trajectory of disease.

Naglazyme (galsulfase) may not be approved when the above criteria are not met and for all other indications.

Quantity Limits

Naglazyme (galsulfase) Quantity Limit

• Naglazyme (galsulfase) 5 mg vial
• Drug
• 1 mg/kg once a week
• Limit
• 1

Coding

The following codes for treatments and procedures applicable to this document are included below for informational purposes. Inclusion or exclusion of a procedure, diagnosis or device code(s) does not constitute or imply member coverage or provider reimbursement policy. Please refer to the member's contract benefits in effect at the time of service to determine coverage or non-coverage of these services as it applies to an individual member.

HCPCS

• J1458
• S9357

ICD-10 Diagnosis

• E76.29
• E76.3

1. Injection, galsulfase, 1 mg [Naglazyme]
2. Home infusion therapy, enzyme replacement intravenous therapy, (e.g., Imiglucerase); administrative services, professional pharmacy services, care coordination, and all necessary supplies and equipment (drugs and nursing visits coded separately), per diem
3. Other mucopolysaccharidoses (includes Maroteaux-Lamy syndrome)
4. Mucopolysaccharidosis, unspecified

Document History

Revised: 9/11/2023