Subject: Aldurazyme 2049-A SGM P2023
Policy:
I. 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 Indications
Aldurazyme is indicated for 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 risks and benefits of treating mildly affected patients
with the Scheie form have not been established.
• Aldurazyme has not been evaluated for effects on the central
nervous system manifestations of the disorder.
All other indications are considered experimental/investigational
and not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate
the prior authorization review:
A. Initial requests: alpha-L-iduronidase enzyme assay and/or
genetic testing results supporting diagnosis.
B. Continuation requests: chart notes documenting a clinically
positive response to therapy, which shall include
improvement, stabilization, or slowing of disease
progression.
III. CRITERIA FOR INITIAL APPROVAL
Mucopolysaccharidosis I (MPS I)
Authorization of 12 months may be granted for treatment of
MPS I when both of the following criteria are met:
A. Diagnosis of MPS I was confirmed by enzyme assay
demonstrating a deficiency of alpha-L-iduronidase enzyme
activity and/or by genetic testing.
B. Member has the Hurler (i.e severe MPS I) or Hurler-Scheie
(i.e. attenuated MPS I) OR the member has 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).
IV. CONTINUATION OF THERAPY
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization for
mucopolysaccharidosis I (MPS I) 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 2019.
- 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 2021 Feb 25]. In: Adam MP, Everman DB, Mirzaa GM, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2023. Accessed Jan 5, 2022
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.
June 18, 2023
