Subject: Vimizim 2057-A SGM P2023

Drug

VIMIZIM  (elosulfase alfa)

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
          Vimizim is indicated for patients with Mucopolysaccharidosis type
          IVA (MPS IVA, Morquio A syndrome).

          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: N-acetylgalactosamine 6-sulfatase enzyme
               assay 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 IVA (MPS IVA)
          Authorization of 12 months may be granted for treatment of
          MPS IVA when the diagnosis of MPS IVA was confirmed by
          enzyme assay demonstrating a deficiency of N-
          acetylgalactosamine 6-sulfatase enzyme activity or by genetic
          testing.

   IV.  CONTINUATION OF THERAPY

          Authorization of 12 months may be granted for continued
          treatment in members requesting reauthorization for
          mucopolysaccharidosis type IVA (MPS IVA, Morquio A syndrome)
          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:

1. Vimizim [package insert]. Novato, CA: BioMarin Pharmaceutical Inc.; December 2019.
2. Hendriksz CJ, Berger KI, Giugliani R, et al. International guidelines for the management and treatment of Morquio A syndrome. Am J Med Genet A. 2015;167A(1):11-25.

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