CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Aldurazyme (laronidase) IV enzyme replacement for MPS I

Policy governing coverage, dosing limits, authorization criteria, renewal criteria, billing codes, and documentation requirements for intravenous Aldurazyme (laronidase) for Medicaid, Commercial, and Medicare members of Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyAldurazyme (laronidase) IV enzyme replacement for MPS I

Policy CodePolicy N/A

Change TypeClarified (references/review dates)

Effective DateFeb 1, 2020

Next Review Date

Key ActionInitial authorization provided for 12 months; may be renewed if renewal criteria met (documentation of uGAG reduction and clinical benefit).

SourceLink

POLICY UPDATE CHANGES

Review dates updated through 05/21/2025; evidence section cites Aldurazyme package insert October 2024 accessed May 2025.

12 monthsInitial authorization length

2668Max HCPCS units per 28 days

0.58 mg/kg weeklyStandard dosing

3Covered ICD-10 codes

Coverage Summary

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Coverage stance: covered_with_criteria. Scope: This policy governs intravenous Aldurazyme (laronidase) for treatment of Mucopolysaccharidosis I (MPS I) for Medicaid, Commercial, and Medicare members. The policy applies to IV Aldurazyme (laronidase) administered per dosing guidance and HCPCS/NDC billing described in the policy. Effective date: 02/01/2020. Last review: 05/21/2025.

Key thresholds and quick facts

Initial authorization length12 months

Max units per 28 days2668 billable units every 28 days

Standard dosing0.58 mg/kg administered once weekly IV

Dose threshold0.58 mg/kg once weekly

Max units threshold2668 units per 28 days

Initial Therapy Criteria

Initial Approval Criteria

Coverage is provided when ALL of the following are met:

ALL of the following

Patient is 6 months of age or older
Therapy is being used to treat non-central nervous system manifestations of the disease and patient does not have severe, irreversible cognitive impairment
Documented baseline value for urinary glycosaminoglycan (uGAG) has been obtained
ONE of the following age-appropriate baseline assessments
- Patients 6 years or greater: percent predicted forced vital capacity (FVC), 6-minute walk test, joint range of motion, left ventricular hypertrophy, growth, quality of life (CHAQ/HAQ/MPS HAQ)
- Patients 6 months to less than 6 years: cardiac status, upper airway obstruction during sleep, growth velocity, mental development, FVC, and/or 6-minute walk test
Definitive diagnosis of MPS I (must meet one of the following)
- Detection of biallelic pathogenic mutations in the IDUA gene by molecular genetic testing
- Detection of deficient activity of the lysosomal enzyme α-L-iduronidase (IDUA)
ONE of the following clinical diagnoses
- Hurler (severe) or Hurler-Scheie (attenuated) forms of disease
- Scheie (attenuated) form of disease with moderate to severe symptoms

Continuation / Renewal Criteria

Renewal Criteria

Authorizations can be renewed when ALL of the following are met:

ALL of the following

Patient continues to meet indication-specific relevant criteria identified in section III (Initial criteria)
Absence of unacceptable toxicity from the drug (examples: anaphylaxis, severe hypersensitivity reactions, acute respiratory complications, acute cardiorespiratory failure, severe infusion reactions)
Patient has a documented reduction in uGAG levels compared to pretreatment baseline
ONE of the following demonstration of clinical benefit (age-specific)
- Patients 6 years or greater: stability or improvement in percent predicted FVC and/or 6-minute walk test, increased joint range of motion, decreased left ventricular hypertrophy, improved growth, improved quality of life (clinically meaningful change in the CHAQ/HAQ/MPS HAQ disability index)
- Patients 6 months to less than 6 years: stability or improvement in cardiac status, upper airway obstruction during sleep, growth velocity, mental development, FVC and/or 6-minute walk test

Coverage Limitations / Exclusions

Exclusions and limits: Therapy is covered only for non-central nervous system manifestations of MPS I and is not provided for patients with severe, irreversible cognitive impairment. Claims may be denied when disease affects only the CNS or when severe, irreversible cognitive impairment is present. Implicit exclusion: CNS-only indications are not covered under this policy.

Provider Actions & Billing Rules

Prior Authorization

Authorization duration and renewal

Initial authorization provided for 12 months. Authorization may be renewed when renewal criteria are met (see renewal requirements).

Documentation Required

Baseline testing required

Obtain and document a baseline urinary glycosaminoglycan (uGAG) value and age-appropriate baseline assessments (e.g., percent predicted FVC, 6MWT, joint range of motion, cardiac status, growth, mental development, quality of life measures) prior to initiating therapy.

Documentation Required

Diagnosis confirmation

Document definitive diagnosis of MPS I by either detection of biallelic pathogenic IDUA mutations by molecular genetic testing or by demonstration of deficient α-L-iduronidase (IDUA) enzyme activity.

Denial Risk

Coverage limited to non-CNS manifestations

Therapy is covered only for non-central nervous system (non-CNS) manifestations. Claims may be denied if used for CNS-only disease or in patients with severe, irreversible cognitive impairment.

Billing Rule

HCPCS unit reporting

Report HCPCS code J1931. One billable unit = 0.1 mg of laronidase. Adhere to the maximum of 2668 billable units every 28 days.

Clinical Evidence Summary

182 wksOpen-label extension: sustained functional improvements and continued urinary GAG reduction

Evidence summary: Approval is supported by a 26-week randomized, double-blind, placebo-controlled trial demonstrating improvement in forced vital capacity (0.58 mg/kg IV weekly; FVC p=0.02) and a 38-meter greater 6-minute walk distance versus placebo, with all trial completers entering a 182-week open-label extension showing sustained functional improvements and reduced urinary GAGs. Reported adverse reactions include infusion-related events such as rash, pyrexia, headache, and flushing, and life-threatening hypersensitivity reactions including anaphylaxis. The policy references the Aldurazyme package insert (October 2024) as a source for safety and prescribing information.

Background

Background: Aldurazyme (laronidase) is an FDA-approved enzyme replacement therapy indicated for treatment of non-central nervous system manifestations of MPS I, including Hurler, Hurler-Scheie, and Scheie forms. Mechanism: replacement of the deficient lysosomal enzyme α-L-iduronidase (IDUA). Known safety risks include infusion-related reactions and anaphylaxis.

Definitions

uGAGUrinary glycosaminoglycan

FVCForced vital capacity

6MWT6-minute walk test

Revision History

2025-05-21reviewLatest

No clinical policy statement changes indicated in document; assumes routine updates to references and review dates. Review dates updated through 05/21/2025; evidence section cites Aldurazyme package insert October 2024 accessed May 2025.

2020-02-01effective

Policy effective date established.