Subject: Emflaza 1636-A SGM P2023

Drug

EMFLAZA  (deflazacort)

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 Indication
          Emflaza is indicated for the treatment of Duchenne muscular
          dystrophy (DMD) in patients 2 years of age and older.

          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.  Laboratory confirmation of DMD diagnosis by genetic testing
               or muscle biopsy
          B.  Chart documentation of weight gain/obesity or persistent
               psychiatric/behavioral issues with previous prednisone or
               prednisolone treatment.


  III.  CRITERIA FOR INITIAL APPROVAL
          Duchenne Muscular Dystrophy
            Authorization of 6 months may be granted for treatment of DMD
          when all of the following criteria are met:
          A.  The diagnosis of DMD was confirmed by one of the following
               criteria:
               1.  Genetic testing demonstrating a mutation in the DMD
               2.  Muscle biopsy demonstrating absent dystrophin.
          B. The member is 2 years of age or older.
          C.  The member has tried prednisone or prednisolone and
               experienced unmanageable and clinically significant weight
               gain/obesity or psychiatric/behavioral issues (e.g., abnormal
               behavior, aggression, irritability):
               1.  For weight gain/obesity: body mass index is in the
                    overweight or obese category while receiving treatment
                    with prednisone or prednisolone (refer to Appendix for
                    weight status categories for children and adults).
               2.  For psychiatric/behavioral issues: psychiatric/behavioral
                    issues persisted beyond the first 6 weeks of treatment
                    with prednisone or prednisolone.


   IV.  CONTINUATION OF THERAPY

          Authorization of 12 months may be granted for members
          requesting continuation of therapy when all of the following
          criteria are met:
          A.  The member meets all initial authorization criteria.
          B.  The member is receiving a clinical benefit from Emflaza
               therapy, such as improvement or stabilization of muscle
               strength or pulmonary function.


     V.  APPENDIX

  Body Mass Index Percentile and Weight Status Category for
  Children 2 Through 19 Years of Age

Body Mass Index Percentile Range	Weight Status
Less than the 5th percentile	Underweight
5th percentile to less than the 85th percentile	Healthy Weight
85th to less than the 95th percentile	Overweight
Equal to or greater than the 95th percentile	Obese

 

Body Mass Index and Weight Status Category for Adults (20 Years of Age and Older)

Body Mass Index	Weight Status
Below 18.5	Underweight
18.5 – 24.9	Healthy Weight
25.0 – 29.9	Overweight
30.0 and Above	Obese

Place of Service:

Outpatient

The above policy is based on the following references:

1. Emflaza [package insert]. South Plainfield, NJ: PTC Therapeutics, Inc.; June 2021.
2. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis and pharmacological and psychosocial management. Lancet Neurol. 2010;9:77-93.
3. Gloss D, Moxley RT, Ashwal S, Oskoui M. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology. 2016;86(5):465-472.
4. Griggs RC, Miller JP, Greenberg CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016;87(20):2123-2131.
5. Centers for Disease Control and Prevention. Assessing Your Weight. https://www.cdc.gov/healthyweight/assessing/bmi/ Accessed February 21, 2023. 
6. Birnkrant DJ, Bushby, K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251-267.

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.

October 22, 2023