Subject: Vimizim 2057-A SGM P2022

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 26, 2022