Subject: VPRIV 2058-A SGM P2024

Drug

VPRIV  (velaglucerase 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.

          A.  FDA-Approved Indication
               VPRIV is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1
               Gaucher disease.

          B.  Compendial Uses
               1.  Gaucher disease type 2
               2.  Gaucher disease type 3

          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:
          beta-glucocerebrosidase enzyme assay or genetic testing results supporting diagnosis.


  III.   CRITERIA FOR INITIAL APPROVAL

          A.  Gaucher disease type 1
               Authorization of 12 months may be granted for treatment of Gaucher disease type 1 when
               the diagnosis of Gaucher disease was confirmed by enzyme assay demonstrating a
               deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic testing.

          B.  Gaucher disease type 2
               Authorization of 12 months may be granted for treatment of Gaucher disease type 2 when
               the diagnosis of Gaucher disease was confirmed by enzyme assay demonstrating a
               deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic testing.

          C.  Gaucher disease type 3
               Authorization of 12 months may be granted for treatment of Gaucher disease type 3 when
               the diagnosis of Gaucher disease was confirmed by enzyme assay demonstrating a
               deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic testing.


   IV.  CONTINUATION OF THERAPY

          Authorization of 12 months may be granted for continued treatment of an indication listed in
          Section III when all of the following criteria are met:
          A.  Member meets the criteria for initial approval.
          B.  Member is not experiencing an inadequate response or any intolerable adverse events from
               therapy.

 

Place of Service:

Outpatient

The above policy is based on the following references:

1. VPRIV [package insert]. Lexington, MA: Takeda Pharmaceuticals U.S.A., Inc.; September 2021.
2. Pastores GM, Hughes DA. Gaucher Disease. 2000 July 27 [Updated March 9, 2023]. In: Adam MP, Everman DB, Mirzaa GM, et al, editors. GeneReviews® [Internet]. Seattle, WA: University of Washington, Seattle; 1993-2023.
3. Kaplan P, Baris H, De Meirleir L, et al. Revised recommendations for the management of Gaucher disease in children. Eur J Pediatr. 2013;172:447-458.
4. Vellodi A, Tylki-Szymanska A, Davies EH, et al. Management of neuronopathic Gaucher disease: revised recommendations. European Working Group on Gaucher Disease. J Inherit Metab Dis. 2009;32(5):660.
5. National Organization for Rare Disorders. (2003). NORD guide to rare disorders. Philadelphia: Lippincott Williams & Wilkins.

 

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 16, 2024