CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Vimizim (elosulfase alfa) (Intravenous)

Policy governs coverage, dosing limits, initial and renewal medical necessity criteria, dosing/administration, billing codes, and quantity limits for Vimizim (elosulfase alfa) for Medicaid, Commercial, and Medicare-Medicaid Plan (MMP) members.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyVimizim (elosulfase alfa) (Intravenous)

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJan 1, 2020

Next Review Date

Key ActionSubmit documentation of diagnosis confirmation (enzyme assay or GALNS genetic testing) and baseline measures (endurance test, pulmonary function test, or urine KS/GAG) to support medical necessity.

POLICY UPDATE CHANGES

No material changes: has_material_change = false

1Covered diagnosis

2 mg/kg weeklyStandard dosing

230 mg/weekMax billed per 7 days

184 vials/28 daysNDC quantity limit

Coverage Summary

Coverage stance: covered_with_criteria for Vimizim (elosulfase alfa) for the treatment of Morquio A syndrome (MPS IVA). Scope: policy governs coverage, dosing limits, initial and renewal medical necessity criteria, dosing/administration, billing codes, and quantity limits for Medicaid, Commercial, and Medicare-Medicaid Plan (MMP) members. Effective date: 01/01/2020. Last review date: 01/15/2025. Patient population: individuals with a diagnosis of Morquio A syndrome (MPS IVA).

Initial Approval Criteria — Mucopolysaccharidosis Type IVA (MPS IVA, Morquio A Syndrome)

Coverage is provided when ALL of the following are met:

ALL of the following

Documented diagnosis of Mucopolysaccharidosis type IVA
Confirmation of diagnosis by ONE of the following
- Absence or marked reduction in N-acetylgalactosamine 6-sulfatase (GALNS) enzyme activity in cultured fibroblasts or leukocytes
- Detection of biallelic pathogenic mutations in the GALNS gene by genetic molecular testing (e.g., sequence analysis and/or deletion/duplication analysis)
ANY of the following
- Endurance test (e.g., Six-minute walk test [6-MWT], timed 25-foot walk test [T25FW], three-minute stair climb test [3-MSCT])
- Pulmonary function tests (e.g., maximum voluntary ventilation [MVV], percent predicted forced vital capacity [FVC])
- Urine keratan sulfate (KS) or urine glycosaminoglycan (GAG) levels

Renewal Criteria

Approval may be renewed when ALL of the following are met:

ALL of the following

Patient continues to meet Universal and indication-specific criteria listed in initial approval
Absence of unacceptable toxicity (examples include anaphylaxis, hypersensitivity reactions, acute respiratory complications, spinal/cervical cord compression)
ANY of the following
- Stability or improvement in endurance tests (6-MWT, T25FW, 3-MSCT)
- Stability or improvement in pulmonary function tests (e.g., FVC)
- Stability or reduction in urine keratan sulfate (KS) or urine glycosaminoglycan (GAG)

MMP Exception

Additional rule for Medicare-Medicaid Plan members:

ALL of the following

MMP members who have previously received this medication within the past 365 days are not subject to Therapy Requirements: Step

Applicable Codes

HCPCS code for billingHCPCSCovered

J1322

Injection, elosulfase alfa, 1 mg (1 billable unit = 1 mg)

NDC / product identifierNDC

68135-0100-xx

Vimizim 5 mg/5 mL injection (product NDC pattern listed)

Covered diagnosis codesICD-10Covered

E76.210

Morquio A mucopolysaccharidoses

Billing unit guidance: HCPCS code J1322 is billed per mg (1 billable unit = 1 mg). Do not exceed 230 billable units (230 mg) every 7 days. NDC/product quantity limit: 184 vials every 28 days.

Provider Actions & Billing Rules

Prior Authorization

Prior authorization and initial documentation

Submit documentation of diagnosis confirmation (absence or marked reduction in GALNS enzyme activity in cultured fibroblasts or leukocytes OR detection of biallelic pathogenic mutations in the GALNS gene) and baseline measures (endurance test, pulmonary function test, or urine KS/GAG) to support medical necessity.

Affected codes: J1322
Affected codes: E76.210

Documentation Required

Baseline and ongoing objective measures

Provide baseline and follow-up measures of endurance (e.g., 6-MWT, T25FW, 3-MSCT), pulmonary function (e.g., FVC, MVV), or urine biomarkers (urine keratan sulfate or urine GAG) to document response for renewal (stability or improvement compared to baseline).

Billing Rule

Dose and quantity limits enforcement

Billing and quantity limits are enforced as follows.

Bill J1322 per mg (1 unit = 1 mg)
Do not exceed 230 mg every 7 days
NDC quantity limit of 184 vials every 28 days

Denial Risk

Denial risk for unacceptable toxicity

Requests may be denied or discontinued for unacceptable toxicity. Examples include anaphylaxis, hypersensitivity reactions, acute respiratory complications, and spinal/cervical cord compression.

Clinical Evidence

Improved 6-minute walk test distancePhase 3 trials — endurance (6-MWT) improvement

Improved FVC and MVVPhase 3 trials — pulmonary function (FVC, MVV) improvement

Background evidence summary: Elosulfase alfa is approved as enzyme replacement therapy for Morquio A (MPS IVA). Phase 3 trials demonstrated improvements in endurance (6-minute walk test) and respiratory function (forced vital capacity and maximum voluntary ventilation). Commonly reported adverse events include infusion-related reactions, fever, and headache.

Background & Definitions

Elosulfase alfa (Vimizim) is an enzyme replacement therapy that replaces the deficient lysosomal enzyme in patients with Morquio A syndrome (MPS IVA). It is administered as 2 mg/kg IV once weekly. Safety profile includes risk of infusion-related reactions and other adverse events such as fever and headache; unacceptable toxicities (e.g., anaphylaxis, severe hypersensitivity, acute respiratory complications, spinal/cervical cord compression) may lead to denial or discontinuation. Definitions: MPS IVA = Mucopolysaccharidosis type IVA (Morquio A Syndrome); 6-MWT = Six-minute walk test.

Key dosing and eligibility thresholds

Age>= 5 years

Dose per administration2 mg/kg IV once weekly

Max units230 billable units (230 mg) every 7 days

Quantity limit184 vials every 28 days

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyVimizim (elosulfase alfa) (Intravenous)

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJan 1, 2020

Next Review Date

On This Page

OpenPayer

Vimizim (elosulfase alfa) (Intravenous)

Coverage Summary

Applicable Codes

Provider Actions & Billing Rules

Clinical Evidence

Background & Definitions

Revision History