CurrentHealth NetPolicy CP PHAR.152

Clinical Policy: Laronidase (Aldurazyme)

Clinical policy governing coverage and prior authorization criteria for laronidase (Aldurazyme) for treatment of mucopolysaccharidosis I (MPS I) across Commercial, HIM, and Medicaid lines of business administered by Centene-affiliated health plans.

Policy Summary

PayerHealth Net

PolicyClinical Policy: Laronidase (Aldurazyme)

Policy CodePolicy CP PHAR.152

Change TypeAnnual reviews; clarifications and documentation requirement added

Effective DateFeb 1, 2016

Next Review Date

Key ActionProvider must submit documentation supporting that the member meets all approval criteria including enzyme assay or DNA testing, current weight, diagnosis subtype, and symptom severity for Scheie form.

POLICY UPDATE CHANGES

2Q 2021 annual review: clarified the covered subtypes of MPS I to align with the FDA-approved indication.

2Q 2022 annual review: added requirement for documentation of member's current weight for dose calculation purposes; references reviewed and updated.

Annual reviews 2023-2025: no significant changes; references reviewed and updated.

3Covered MPS I subtypes

0.58Max weekly dose (mg/kg)

6 moMedicaid/HIM min age

J1931Primary HCPCS code

Coverage Summary

Coverage stance: covered with criteria for laronidase (Aldurazyme) for treatment of mucopolysaccharidosis I (MPS I). The policy covers the FDA‑approved subtypes: Hurler and Hurler‑Scheie forms, and the Scheie form with moderate to severe symptoms. This clinical policy applies across lines of business including Commercial, HIM/Marketplace, and Medicaid. The therapy carries a boxed warning for risk of life‑threatening hypersensitivity reactions, including anaphylaxis. Aldurazyme has not been evaluated for effects on central nervous system (CNS) manifestations of MPS I.

Key Coverage Thresholds

Maximum dosing0.58 mg/kg per week (rounded up to the nearest whole vial)

Medicaid/HIM minimum age6 months

Primary HCPCS codeJ1931

Initial Therapy Criteria

Provider must submit documentation supporting that member has met all approval criteria.

ALL of the following

Diagnosis of MPS I confirmed by one of the following
- Enzyme assay demonstrating deficiency of alpha-L-iduronidase activity.
- DNA testing consistent with MPS I.
Mucopolysaccharidosis I: Hurler, Hurler-Scheie, and Scheie Forms (must meet all)
- Hurler or Hurler-Scheie form of MPS I.
- Scheie form of MPS I with moderate to severe symptoms.
Documentation of member's current weight (in kg).
Age: member is at least 26 months for Commercial; Medicaid/HIM minimum 6 months.
Medicaid/HIM minimum age: 6 months.
Dose does not exceed 0.58 mg/kg per week (rounded up to the nearest whole vial).<= 0.58 mg/kg per week

Unproven / Exclusions

Other / Not Authorized Diagnoses and Indications

Coverage not authorized unless referenced policies or off-label policies are met.

Other diagnoses/indications - pathways: For drugs on formulary/PDL: follow the no coverage criteria policy for the relevant line of business (CP.CPA.190 for commercial, HIM.PA.33 for marketplace, CP.PMN.255 for Medicaid); For drugs NOT on formulary/PDL: follow the non-formulary policy (CP.CPA.190 for commercial, HIM.PA.103 for marketplace, CP.PMN.16 for Medicaid); If requested use is not listed and initial criteria do not apply: refer to off-label use policy (CPCPA.09 commercial, HIMPA.154 marketplace, CPPMN.53 Medicaid)

Diagnoses/Indications Not Authorized: Non-FDA approved indications not addressed in this policy are not authorized unless there is sufficient documentation of efficacy and safety per off-label use policies (CP.CPA.09 commercial, HIM.PA.154 marketplace, CP.PMN.53 Medicaid) or applicable evidence of coverage documents

Provider Actions / Prior Authorization

Prior Authorization

Prior authorization and documentation

Provider must submit documentation (office chart notes, lab results, or other clinical information) supporting enzyme assay or DNA testing, current weight, diagnosis subtype, and symptom severity for Scheie form when requesting prior authorization for Aldurazyme.

J1931

Documentation Required

Weight documentation for dosing

Provider must document the member's current weight in kilograms for dose calculation. The dose must not exceed 0.58 mg/kg per week (rounded up to the nearest whole vial).

Denial Risk

Coverage exclusion for non-FDA indications

Requests for non‑FDA approved indications that are not addressed in this policy will be denied unless there is sufficient off‑label evidence or the request meets the referenced policy criteria.

Off-label use policy codes: CPCPA.09, HIMPA.154, CPPMN.53
Formulary/no-coverage & non-formulary policy codes: CP.CPA.190, HIM.PA.33, CP.PMN.255, HIM.PA.103, CP.PMN.16

Clinical Evidence

10%relative improvement in percent predicted FVC vs baseline (patients ≥6 yrs)

19.7 mmean increase in 6MWT after 26 weeks (patients ≥6 yrs)

The evidence cited in the policy comes from clinical trials referenced in the policy documents. Trial outcomes reported for patients ≥6 years include improvements in pulmonary function and exercise capacity (percent predicted FVC and 6‑minute walk test), and the specific references are listed in the policy.

Background

Background: Laronidase (Aldurazyme) is a recombinant alpha‑L‑iduronidase enzyme indicated for treatment of the Hurler and Hurler‑Scheie forms of MPS I and for the Scheie form of MPS I in patients with moderate to severe symptoms. The product labeling and policy include a boxed warning for life‑threatening hypersensitivity and anaphylaxis. Aldurazyme has not been evaluated for effects on central nervous system manifestations of the disorder.

Definitions

6MWT6-minute walk test

FVCForced vital capacity

MPS IMucopolysaccharidosis I

Revision History

05.21.2021P&T approval

P&T approval noted; 2Q 2021 annual review clarified the covered subtypes of MPS I to align with the FDA-approved indication (Hurler, Hurler-Scheie, and Scheie with moderate-to-severe symptoms).

05.22.2022P&T approval

P&T approval noted; 2Q 2022 annual review added requirement for documentation of the member's current weight for dose calculation purposes and references were reviewed/updated.

05.25.2025P&T approvalLatest

Most recent P&T approval date; 2Q 2023–2Q 2025 annual reviews reported no significant changes and references were reviewed/updated (annual reviews 2023–2025).