Subject: miglustat products 2098-A SGM P2024

Drug

ZAVESCA  (miglustat)

YARGESA  (miglustat)

miglustat

OPFOLDA  (miglustat)

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 Indications
               1.  miglustat (generic)/Yargesa/Zavesca:
                    Indicated as monotherapy for the treatment of adult patients with mild to moderate type 1
                    Gaucher disease for whom enzyme replacement therapy is not a therapeutic option
                    (e.g., due to allergy, hypersensitivity, or poor venous access).

               2.  Opfolda:
                    Indicated, in combination with Pombiliti, for the treatment of adult patients with late-onset
                    Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing greater
                    than or equal to 40 kg and who are not improving on their current enzyme replacement
                    therapy (ERT).

               B.  Compendial Uses
                    Niemann-Pick disease, type C

               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.  Gaucher disease type 1: beta-glucocerebrosidase enzyme assay or genetic testing results
               supporting diagnosis.
          B.  Niemann-Pick disease, type C: genetic testing results showing mutations in NPC1 or NPC2
          C.  Late-onset Pompe disease:
               1.  Initial requests: acid alpha-glucosidase enzyme assay or genetic testing results
                    supporting diagnosis.
               2.  Continuation requests: chart notes documenting a positive response to therapy (e.g.,
                    improvement, stabilization, or slowing of disease progression for motor function, walking
                    capacity, respiratory function, muscle strength).


   III.  CRITERIA FOR INITIAL APPROVAL

          A.  Gaucher disease type 1 (miglustat (generic)/Yargesa/Zavesca only)
               Authorization of 12 months may be granted for treatment of Gaucher disease type 1 when
               ALL of the following criteria are met:
               1.  The diagnosis of Gaucher disease was confirmed by enzyme assay demonstrating a
                    deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic
                    testing, and
               2.  The member has a documented inadequate response to, intolerable adverse events
                    with, or a clinical reason to not use enzyme replacement therapy (e.g., allergy,
                    hypersensitivity, poor venous access).

          B.  Niemann-Pick disease, type C (miglustat (generic)/Yargesa/Zavesca only)
               Authorization of 12 months may be granted for treatment of Niemann-Pick disease, type C
               when the diagnosis was confirmed by genetic testing results showing mutations in NPC1 or
               NPC2 genes.

          C.  Late-onset Pompe disease (Opfolda only)
               Authorization of 12 months may be granted for treatment of late-onset Pompe disease when
               all of the following criteria are met:
               1.  Member is 18 years of age or older.
               2.  Member weighs greater than or equal to 40 kg.
               3.  Diagnosis was confirmed by enzyme assay demonstrating a deficiency of acid alpha-
                    glucosidase enzyme activity or by genetic testing.
               4.  Member is not improving on current enzyme replacement therapy (ERT) (e.g.,
                    Lumazine, Nexviazyme).
               5.  The requested medication will be taken in combination with Pombiliti (cipaglucosidase
                    alfa-atga).


   IV.  CONTINUATION OF THERAPY

          A.  Gaucher disease type 1 (miglustat (generic)/Yargesa/Zavesca only)
               Authorization of 12 months may be granted for continued treatment in members requesting
               reauthorization for Gaucher disease type 1 when all of the following criteria are met:
               1.  The diagnosis of Gaucher disease was confirmed by enzyme assay demonstrating a
                    deficiency of beta-glucocerebrosidase (glucosidase) enzyme activity or by genetic
                    testing.
               2.  Member is not experiencing an inadequate response or any intolerable adverse events
                    from therapy.

          B.  Niemann-Pick disease, type C (miglustat (generic)/Yargesa/Zavesca only)
                Authorization of 12 months may be granted for continued treatment in members requesting
                reauthorization for Niemann-Pick disease, type C when all of the following criteria are met:
               1.  Member meets the criteria for initial approval.
               2.  Member is not experiencing an inadequate response or any intolerable adverse events
                    from therapy.

          C.  Late-onset Pompe disease (Opfolda only)
               Authorization of 12 months may be granted for continued treatment in members requesting
               reauthorization for late-onset Pompe disease when both of the following criteria are met:
               1.  Member is responding to therapy (e.g., improvement, stabilization, or slowing of disease
                    progression for motor function, walking capacity, respiratory function, or muscle
                    strength).
               2.  The requested medication will be taken in combination with Pombiliti (cipaglucosidase
                    alfa-atga).

 

Place of Service:

Outpatient

The above policy is based on the following references:

1. Zavesca [package insert]. Titusville, NJ: Actelion Pharmaceuticals US, Inc.; August 2022.
2. miglustat [package insert]. Titusville, NJ: CoTherix, Inc.; December 2022.
3. Lexicomp Online, Lexi-Drugs Online. Waltham, MA: UpToDate, Inc.; Updated November 24, 2023. https://online.lexi.com. Accessed December 6, 2023.
4. National Organization for Rare Disorders. (2003). NORD guide to rare disorders. Philadelphia: Lippincott Williams & Wilkins.
5. Opfolda [package insert]. Philadelphia, PA: Amicus Therapeutics US, LLC; September 2023
6. Yargesa [package insert]. Parsippany, NJ: Edenbridge Pharmaceuticals, LLC; January 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 16, 2024