CurrentEmblemHealthPolicy MG.MM.PH.305

Medical Policy: Naglazyme® (galsulfase) intravenous infusion

Defines medical necessity criteria, dosing limits, authorization length, renewal criteria, applicable procedure/NDC/ICD-10 codes, and provider requirements for coverage of intravenous Naglazyme for Mucopolysaccharidosis Type VI (MPS VI).

Policy Summary

PayerEmblemHealth

PolicyMedical Policy: Naglazyme® (galsulfase) intravenous infusion

Policy CodePolicy MG.MM.PH.305

Change TypeMaterial criteria revisions and additions (initial criteria, age/baseline testing, renewal, prescribing specialty)

Effective DateMar 17, 2025

Next Review Date

Key ActionThe treating physician or primary care provider must submit clinical evidence (diagnostic tests, baseline functional tests, uGAG) to EmblemHealth for preauthorization or post-payment review.

SourceLink

POLICY UPDATE CHANGES

Replaced 'arylsulfatase B gene mutation' language with 'biallelic pathogenic or likely pathogenic arylsulfatase B (ARSB) gene variants' in Initial Criteria.

Added requirement that patient is at least 5 years of age and baseline functional tests (12-MWT, 3-MSCT, and/or PFTs) and baseline uGAG are documented.

Added Renewal Criteria including absence of unacceptable toxicity, documented reduction in uGAG, and demonstration of beneficial disease response (improvement or stability in 12-MWT, 3-MSCT, or PFTs).

Specified prescribing provider specialties (geneticist, endocrinologist, metabolic disorder subspecialist, or lysosomal storage disorder specialist) for initiation.

1Covered Indication (MPS VI)

115Max HCPCS units per 7 days

>=5Minimum age (years)

1 mg/kgMax dose per administration

Coverage Summary

Covered with criteria. Defines medical necessity criteria, dosing limits, authorization length, renewal criteria, applicable procedure/NDC/ICD-10 codes, and provider requirements for intravenous Naglazyme (galsulfase) for Mucopolysaccharidosis Type VI (MPS VI). Effective date: 2025-03-17. Policy number: MG.MM.PH.305. Key scope items include minimum age >= 5 years, maximum dose per administration <= 1 mg/kg, and maximum billing of 115 billable units every 7 days.

Initial Therapy Criteria

Initial Criteria

Approve if the patient meets the following criteria:

ALL of the following

Diagnosis established by ONE of the following
- Patient has a laboratory test demonstrating deficient N-acetylgalactosamine 4-sulfatase (arylsulfatase B) activity in leukocytes or fibroblasts
- Patient has a molecular genetic test demonstrating biallelic pathogenic or likely pathogenic arylsulfatase B (ARSB) gene variants
Patient is at least 5 years of age>= 5 years
Documented baseline 12-minute walk test (12-MWT), 3-minute stair climb test (3-MSCT), and/or pulmonary function tests (e.g., FEV1, etc.)
Documented baseline value for urinary glycosaminoglycan (uGAG)
Therapy is being used to treat non-central nervous system manifestations of the disease and patient does not have severe, irreversible cognitive impairment
Naglazyme is prescribed by or in consultation with a geneticist, endocrinologist, a metabolic disorder subspecialist, or a physician who specializes in the treatment of lysosomal storage disorders

Continuation / Renewal Criteria

Renewal Criteria

Coverage can be renewed based on the following criteria:

ALL of the following

Patient continues to meet Initial Criteria
Absence of unacceptable toxicity from the drug (examples include: anaphylaxis and hypersensitivity reactions, immune-mediated reactions, acute respiratory complications associated with administration, acute cardiorespiratory failure, severe infusion reactions, spinal or cervical cord compression, etc.)
Patient has a documented reduction in uGAG levels compared to pretreatment baseline
Demonstrated beneficial disease response defined by ONE or more of the following

Exclusions / Not Covered Uses

Therapy is not approved for treatment of central nervous system manifestations or for patients with severe, irreversible cognitive impairment.

Applicable Codes

Covered HCPCS / J-codeHCPCSCovered

J1458

Injection, galsulfase, 1 mg

Applicable NDCsNDC

68135-0020-01

Naglazyme 1mg/mL Solution

Applicable ICD-10 DiagnosesICD-10Covered

E76.29

Other mucopolysaccharidoses

Provider Actions & Authorization

Prior Authorization

Preauthorization and clinical documentation required

The treating physician or primary care provider must submit clinical evidence that the member meets the criteria for treatment (diagnostic tests, baseline functional tests, uGAG) to EmblemHealth for preauthorization or post-payment review.

Documentation Required

Baseline and follow-up testing documentation

Document baseline 12-minute walk test (12-MWT), 3-minute stair climb test (3-MSCT) and/or pulmonary function tests and baseline uGAG prior to initiation; for renewal document reduction in uGAG and improvement or stability in 12-MWT, 3-MSCT, or pulmonary function testing.

Clinical Evidence

Evidence includes: Prescribing information, Naglazyme intravenous infusion [prescribing information], BioMarin; April 2020. The brief notes clinical improvements in walking and stair climbing capacity and describes the enzyme mechanism; no quantitative trial metrics beyond baseline functional tests are provided in the policy.

Background

Naglazyme (galsulfase) is a recombinant human N-acetylgalactosamine 4-sulfatase indicated for Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome). The enzyme catalyzes the hydrolysis of the sulfate ester from glycosaminoglycans (chondroitin 4-sulfate and dermatan sulfate), and treatment has been shown to improve walking and stair climbing capacity.

Key thresholds

Age>= 5 years

Max dose per administration<= 1 mg/kg per dose

Administration frequencyNo more frequently than once weekly

Max units (HCPCS unit)115 billable units every 7 days

Definitions

uGAG: Urinary glycosaminoglycan — the measured urinary biomarker used as a baseline and to document reduction on therapy.

12-MWT: 12-minute walk test — a baseline and follow-up functional assessment of walking capacity referenced in initial and renewal criteria.

3-MSCT: 3-minute stair climb test — a baseline and follow-up functional assessment of stair-climbing capacity referenced in initial and renewal criteria.

ARSB: Arylsulfatase B (arylsulfatase B gene) — the enzyme (and gene) deficient in MPS VI; molecular testing for biallelic pathogenic or likely pathogenic ARSB variants is an accepted diagnostic criterion.

Revision History

2025-03-17revisedLatest

Clarified genetic testing requirement: replaced 'arylsulfatase B gene mutation' language with 'biallelic pathogenic or likely pathogenic arylsulfatase B (ARSB) gene variants' in Initial Criteria.

2025-03-17addedLatest

Added baseline testing and age threshold to Initial Criteria: patient must be at least 5 years of age and have documented baseline 12-minute walk test (12-MWT), 3-minute stair climb test (3-MSCT) and/or pulmonary function tests, and a documented baseline urinary glycosaminoglycan (uGAG) value.

Policy Summary

PayerEmblemHealth

PolicyMedical Policy: Naglazyme® (galsulfase) intravenous infusion

Policy CodePolicy MG.MM.PH.305

Change TypeMaterial criteria revisions and additions (initial criteria, age/baseline testing, renewal, prescribing specialty)

Effective DateMar 17, 2025

Next Review Date

SourceLink

On This Page

Improvement in or stability of 12-minute walk test (12-MWT)

Improvement in or stability of 3-minute stair climb test (3-MSCT)

Improvement in or stability of pulmonary function testing (e.g., FEV1, etc.)

Billing Rule

Dosing and billing limits

Each dose must not exceed 1 mg/kg administered intravenously no more frequently than once weekly. Bill using J1458 (Injection, galsulfase, 1 mg) with a maximum of 115 billable HCPCS units every 7 days.

J1458
Max dose per administration: 1 mg/kg
Max units: 115 billable units every 7 days
Frequency: no more frequently than once weekly

Denial Risk

Denial risk for CNS-only indications or severe cognitive impairment

Therapy is not approved for treatment of central nervous system manifestations or for patients with severe, irreversible cognitive impairment.

Formalized Renewal Criteria: coverage renewal requires continued meeting of Initial Criteria, absence of unacceptable toxicity, documented reduction in uGAG compared to pretreatment baseline, and demonstrated beneficial disease response defined by improvement or stability in 12-MWT, 3-MSCT, or pulmonary function testing.

2025-03-17clarifiedLatest

Specified prescribing provider specialties for initiation: Naglazyme must be prescribed by or in consultation with a geneticist, endocrinologist, a metabolic disorder subspecialist, or a physician who specializes in the treatment of lysosomal storage disorders.

2025-03-17clarifiedLatest

Dosing and billing limits reiterated: each dose must not exceed 1 mg/kg IV no more frequently than once weekly and billed using J1458 with a maximum of 115 billable units every 7 days.