Subject: Aldurazyme 2049-A SGM P2022
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).
III. 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.
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 26, 2022
