CurrentUCarePolicy N/A

Aldurazyme

Defines UCare utilization management medical policy for Aldurazyme (laronidase) intravenous infusion for treatment of Mucopolysaccharidosis Type I (MPS I), including required diagnostic criteria, prescriber qualifications, dosing limits, authorization duration, and non-covered situations.

Policy Summary

PayerUCare

PolicyAldurazyme

Policy CodePolicy N/A

Change TypeClarified genetic confirmation languageroutine annual revisions

Effective DateJan 1, 2020

Next Review Date

Key ActionPrior Authorization is recommended; document deficient α-L-iduronidase enzyme activity OR biallelic pathogenic/likely pathogenic IDUA variants, ensure specialist prescriber or consultation, and adhere to dosing limits (≤0.58 mg/kg no more frequently than weekly).

SourceLink

POLICY UPDATE CHANGES

Genetic confirmation language revised to specify 'biallelic pathogenic or likely pathogenic alpha-L-iduronidase gene variants'.

Annual reviews noted with no criteria changes in multiple years; most recent revision date 04/15/2026 with 'No criteria changes.'

1Authorization duration (years)

0.58 mg/kgMax dose per administration

weeklyMax dosing frequency

CwCCoverage stance

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Coverage Summary

Coverage stance: covered_with_criteria. This policy defines UCare utilization management for Aldurazyme (laronidase) intravenous infusion for treatment of Mucopolysaccharidosis Type I (MPS I).

Scope summary: Approvals are for patients with MPS I who meet required diagnostic criteria (either biochemical enzyme deficiency testing or molecular genetic testing demonstrating biallelic pathogenic or likely pathogenic α-L-iduronidase (IDUA) gene variants), and when Aldurazyme is prescribed by or in consultation with an appropriate specialist (geneticist, endocrinologist, metabolic disorder sub-specialist, or a physician who specializes in lysosomal storage disorders).

Dosing limits and authorization duration: Each dose must not exceed 0.58 mg/kg administered intravenously and must be given no more frequently than once weekly. Authorization is for 1 year; extended approvals are allowed if criteria and dosing continue to be met. Requests for doses outside these limits are reviewed case-by-case by a clinician.

Non-covered situations: Coverage is not recommended for circumstances not listed in the Recommended Authorization Criteria; criteria will be updated as new published data become available.

Note: Hematopoietic stem cell transplantation (HSCT) is preferred for severe disease in young children for preservation of neurologic function; Aldurazyme does not cross the blood-brain barrier and is unlikely to improve cognitive function.

Quick Facts

Authorization duration1 year

Maximum single dose0.58 mg/kg per administration

Dosing frequencyNo more frequently than once weekly (IV)

Initial and Continuation Therapy Criteria

FDA-Approved Indication - Initial and Continued Authorization

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

ALL of the following

Diagnosis of Mucopolysaccharidosis Type I (Hurler, Hurler-Scheie, or Scheie syndrome)
Diagnosis established by ONE of the following
- Enzyme assay: Laboratory test demonstrating deficient α-L-iduronidase activity in leukocytes, fibroblasts, plasma, or serum
- Molecular genetic test demonstrating biallelic pathogenic or likely pathogenic α-L-iduronidase (IDUA) gene variants

Provider Actions & Administrative Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is recommended for medical benefit coverage of Aldurazyme. Approvals are provided for 1 year and may be extended if the patient continues to meet the policy criteria and dosing. Requests for doses outside the established dosing in the policy will be reviewed on a case-by-case basis by a clinician.

Documentation Required

Diagnostic Documentation Required

Provider must document either (a) deficient α-L-iduronidase enzyme activity demonstrated in leukocytes, fibroblasts, plasma, or serum, OR (b) molecular genetic testing demonstrating biallelic pathogenic or likely pathogenic α-L-iduronidase (IDUA) gene variants.

Documentation Required

Specialist Prescriber or Consultation Requirement

Applicable Codes

HCPCS / J-code (implied drug billing)HCPCS

No codes listed

Background

Aldurazyme (laronidase) is an enzyme replacement therapy comprising human α-L-iduronidase indicated for MPS I (Hurler, Hurler-Scheie, and Scheie forms).

Pathophysiology: MPS I is an autosomal recessive lysosomal storage disease caused by deficiency of α-L-iduronidase, leading to inability to degrade dermatan and heparan sulfate, accumulation of glycosaminoglycans in lysosomes, and progressive multisystem disease manifesting with characteristic facial features, corneal clouding, cardiomyopathy, hepatosplenomegaly, respiratory insufficiency, skeletal abnormalities, joint stiffness, hernias, and potential cognitive impairment.

HSCT is indicated for severe forms of MPS I in young children (<2 years) who are cognitively intact and has been shown to preserve intellectual development; Aldurazyme does not cross the blood-brain barrier and is therefore unlikely to improve cognitive or neurologic function.

MPS I: Mucopolysaccharidosis type I, a lysosomal storage disease due to α-L-iduronidase deficiency.

Biallelic: Pathogenic or likely pathogenic variants present in both copies of the IDUA gene (i.e., molecular genetic testing demonstrating biallelic pathogenic or likely pathogenic α-L-iduronidase gene variants).

Clinical Evidence

No clinical-evidence trials or detailed evidence entries were supplied in the brief. Clinical trial details (endpoints, outcomes, duration, and safety data) should be added from the full policy, prescribing information, or dossier when available to support comparative effectiveness and benefit-risk assessments.

Revision History

04/24/2024annual_revision

Genetic confirmation language revised to more specifically state 'genetic testing demonstrating biallelic pathogenic or likely pathogenic alpha-L-iduronidase gene variants'.

09/16/2024pt_review

UCare P&T committee reviewed and approved the policy (annual review process).

04/16/2025annual_revision

Annual revision noted: No criteria changes.