Enzyme Replacement Therapy - Aldurazyme (laronidase) Utilization Management Medical Policy
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This policy governs prior authorization, coverage criteria, dosing, and prescribing specialist requirements for Aldurazyme (laronidase) for treatment of Mucopolysaccharidosis type I (MPS I) across Aspirus Medicare Plans and related Aspirus Arise lines.
No material clinical or coverage changes in this revision.
Coverage Criteria for Aldurazyme (laronidase)
FDA-Approved Indication (Initial and Renewal)
Covered when ALL of the following are met:
Requests for doses outside established dosing will be considered case-by-case by a clinician.
Coverage is not recommended for circumstances that are not listed in the Recommended Authorization Criteria. Requests that fall outside the specified criteria may be denied; the criteria will be updated as new published data become available.
Confirmation of MPS I diagnosis by molecular genetic testing when biallelic pathogenic or likely pathogenic IDUA variants are identified
Testing must demonstrate biallelic pathogenic or likely pathogenic variants in IDUA.
Coding and Dosing Limits
| laronidase (Aldurazyme) | HCPCS/brand biologic (documented as Aldurazyme - laronidase intravenous infusion - Genzyme) (no explicit HCPCS/NDC listed in document) |
Provider Actions, Authorization, and Documentation Requirements
Prior authorization required for Aldurazyme (approve 1 year if criteria met)
Prior authorization is recommended for medical benefit coverage of Aldurazyme; approve for 1 year when the patient meets the diagnostic and prescriber criteria. Extended approvals are allowed if the patient continues to meet the Criteria and Dosing. Requests for doses outside the established dosing will be considered case‑by‑case by a clinician.
- Approval duration: 1 year when criteria A and B are met
- Extended approvals permitted if criteria and dosing continue to be met
- Dose exceptions reviewed case‑by‑case by Medical Director or Pharmacist
Relation to alternative therapy (HSCT) — when to use Aldurazyme
Hematopoietic stem cell transplantation (HSCT) is an indicated alternative for severe forms of MPS I in children <2 years who are cognitively intact; Aldurazyme is used when HSCT is not indicated (for children who already have cognitive decline) or is appropriate for older patients.
- HSCT indicated for severe MPS I in children <2 years who are cognitively intact
- Aldurazyme does not cross the blood–brain barrier and may be used in children with prior cognitive decline or in older patients
Required clinical documentation and dosing conformity
Documentation must demonstrate the diagnosis of MPS I by either enzyme assay showing deficient α‑L‑iduronidase activity or by molecular genetic testing showing biallelic pathogenic/likely pathogenic IDUA variants, and must show Aldurazyme is prescribed by or in consultation with an appropriate specialist; dosing requested should conform to the policy's dosing limits.
- Diagnostic evidence: enzyme assay in leukocytes, fibroblasts, plasma, or serum OR molecular genetic test demonstrating biallelic pathogenic/likely pathogenic IDUA variants
- Prescriber: geneticist, endocrinologist, metabolic disorder sub‑specialist, or physician who specializes in lysosomal storage disorders
- Dosing must not exceed 0.58 mg/kg IV no more frequently than once weekly
Denial triggers — requests outside Recommended Authorization Criteria
Coverage is not recommended for requests that fall outside the Recommended Authorization Criteria; such requests may be denied.
- Requests outside the listed Recommended Authorization Criteria are not recommended for approval
- Criteria will be updated as new published data are available
Prescriber requirement — specialist must prescribe or consult
Aldurazyme must be prescribed by or in consultation with a specialist experienced in MPS I (geneticist, endocrinologist, metabolic disorder sub‑specialist, or physician who specializes in lysosomal storage disorders).
- Prescriber requirement is mandatory for approval
- Specialist involvement may be via direct prescription or documented consultation
Definitions and Diagnostic Criteria
Covered Indications
Confirmation of MPS I diagnosis by molecular genetic testing when biallelic pathogenic or likely pathogenic IDUA variants are identified
Testing must demonstrate biallelic pathogenic or likely pathogenic variants in IDUA.
Eligibility Requirements
No top-level eligibility requirements are specified in this policy.
Background on MPS I and Aldurazyme
Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disease caused by deficiency of α-L-iduronidase, leading to accumulation of glycosaminoglycans and progressive multi-organ dysfunction. Clinical features include characteristic facies, corneal clouding, cardiomyopathy, hepatosplenomegaly, respiratory insufficiency, skeletal abnormalities, and cognitive impairment. Definitive diagnosis is by demonstrating deficient α-L-iduronidase activity in leukocytes, fibroblasts, plasma, or serum, or by identifying biallelic pathogenic or likely pathogenic variants in IDUA.
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