Subject: Aldurazyme SGM 2049-A P2025
Policy:
Indications
The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.
FDA-Approved Indications1
Aldurazyme is indicated for the treatment of adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms.
Limitations of Use
- The safety and effectiveness of treating mildly affected patients with the Scheie form have not been established.
- The effect of Aldurazyme on central nervous system manifestations of the disorder has not been determined.
All other indications are considered experimental/investigational and not medically necessary.
Documentation
Submission of the following information is necessary to initiate the prior authorization review:
- Initial requests: alpha-L-iduronidase enzyme assay or genetic testing results supporting diagnosis.
- Continuation requests: chart notes documenting a clinically positive response to therapy, which shall include improvement, stabilization, or slowing of disease progression.
Prescriber Specialties
This medication must be prescribed by or in consultation with a physician who specializes in the treatment of metabolic disease and/or lysosomal storage disorders.
Coverage Criteria
Mucopolysaccharidosis I (MPS I)1-4
Authorization of 12 months may be granted for treatment of MPS I when both of the following criteria are met:
- Diagnosis of MPS I was confirmed by enzyme assay demonstrating a deficiency of alpha-L-iduronidase enzyme activity or by genetic testing.
- Member has one of the following:
- The Hurler form (i.e., severe MPS I).
- The Hurler-Scheie form (i.e., attenuated MPS I).
- The Scheie form (Scheie syndrome; i.e., attenuated MPS I) with moderate to severe symptoms (e.g., normal intelligence, less progressive physical problems, corneal clouding, joint stiffness, valvular heart disease).
Continuation of Therapy
Authorization of 12 months may be granted for continued treatment in members requesting reauthorization for an indication listed in the Coverage Criteria section who have a clinically positive response to therapy, which shall include improvement, stabilization, or slowing of disease progression.
Place of Service:
Outpatient
The above policy is based on the following references:
- Aldurazyme [package insert]. Cambridge, MA: Genzyme Corporation; December 2023.
- Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004;144:581-588.
- Muenzer J, Wraith JE, Clarke LA; International Consensus Panel on Management and Treatment of Mucopolysaccharidosis I. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009 Jan;123(1):19-29.
- Clarke LA. Mucopolysaccharidosis Type I. 2002 Oct 31 [Updated 2024 Apr 11]. In: Adam MP, Everman DB, Mirzaa GM, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2023. Accessed Jan 10, 2025.
Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.
July 22, 2025
