Enzyme Replacement Therapy - Aldurazyme (laronidase) Utilization Management Medical Policy
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Defines prior authorization and coverage criteria for Aldurazyme (laronidase IV) for treatment of mucopolysaccharidosis type I (MPS I) across all UCare plans, including diagnostic requirements, prescriber specialty, dosing limits, and approval duration.
No material clinical or coverage changes in this revision.
Coverage Criteria
Initial therapy
Approve for 1 year if the patient meets BOTH of the following (A and B).
Approval duration: 1 year
Coverage is not recommended for circumstances not listed in the Recommended Authorization Criteria. This policy will not authorize Aldurazyme for uses beyond those explicitly described in the approval criteria and may be updated as new published data become available.
Covered Indications
Diagnosis of MPS I to support Aldurazyme therapy
Molecular genetic testing demonstrating biallelic pathogenic or likely pathogenic IDUA variants is an accepted diagnostic confirmation.
Dosing and Coding
Provider Actions & Authorization
Prior authorization required — 1 year approval when criteria met
Prior authorization is recommended for Aldurazyme; approve for 1 year when the patient meets the diagnostic and prescriber criteria and dosing limits. Extended approvals allowed if criteria and dosing continue to be met; requests for doses outside documented dosing will be considered case-by-case by a clinician.
- Approval duration: 1 year (extensions allowed if criteria/dosing maintained)
- Doses outside policy dosing considered case-by-case by Medical Director or Pharmacist
Step therapy / sequencing — no required prior therapy
No mandatory step therapy or requirement to trial alternative therapies is specified in the policy; hematopoietic stem cell transplantation (HSCT) is noted as indicated for severe early disease in selected patients but is not required prior to Aldurazyme.
- HSCT indicated for severe forms in children <2 years who are cognitively intact; used in selected patients
- Aldurazyme may be appropriate for children <2 years who have cognitive decline or severe physical disease prior to HSCT
Required diagnostic and prescriber documentation
Documentation must demonstrate either (i) deficient α-L-iduronidase activity in leukocytes, fibroblasts, plasma, or serum OR (ii) molecular genetic testing showing biallelic pathogenic or likely pathogenic IDUA variants, and must document that Aldurazyme is prescribed by or in consultation with an appropriate specialist.
- Acceptable diagnostic proof: enzyme assay in leukocytes, fibroblasts, plasma, or serum OR molecular genetic test with biallelic pathogenic/likely pathogenic IDUA variants
- Must document prescribing physician or documented consultation (see specialist requirements)
Prescriber/consultation specialty required
Aldurazyme must be prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder sub-specialist, or a physician who specializes in lysosomal storage disorders.
- Prescriber specialties: geneticist, endocrinologist, metabolic disorder sub-specialist, or lysosomal storage disorder specialist
Denial triggers — non‑listed circumstances not covered
Coverage is not recommended for circumstances not listed in the Recommended Authorization Criteria; requests outside the listed criteria are subject to denial unless documentation meets the policy’s required criteria.
- Denial trigger: absence of required diagnostic confirmation and/or lack of required specialist prescriber/consultation
- Denial trigger: dosing exceeding 0.58 mg/kg or administered more frequently than weekly without case-by-case approval
Definitions
Eligibility Requirements
Eligibility requirements: No additional top-level eligibility nodes are specified beyond the Recommended Authorization Criteria. Eligibility for Aldurazyme is determined by meeting the diagnostic, prescriber, and dosing criteria contained in the Coverage Criteria (see diagnostic confirmation, prescriber specialty, and dosing limits).
Background
Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme α-L-iduronidase, resulting in accumulation of glycosaminoglycans (dermatan and heparan sulfate) and progressive multisystem disease. Clinical severity is variable and described on a spectrum from severe (Hurler) to intermediate (Hurler-Scheie) to mild (Scheie), with manifestations that can include somatic organ dysfunction, airway problems, skeletal abnormalities, and, in severe early-onset disease, cognitive decline.
Treatment options include hematopoietic stem cell transplantation (HSCT) for selected patients with severe early disease to preserve cognition, and enzyme replacement therapy with Aldurazyme (laronidase) to treat somatic manifestations. Aldurazyme does not cross the blood–brain barrier and is therefore unlikely to improve central nervous system or cognitive deficits; its clinical benefit is for peripheral/somatic disease features.
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