EMFLAZA, Deflazacort Form

Deflazacort (Emflaza®) is a corticosteroid. FDA Approved Indication(s) Emflaza is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. Policy/Criteria Provider must submit documentation (such as office chart notes, lab results or other clinical information) supporting that member has met all approval criteria.
It is the policy of health plans affiliated with Centene Corporation® that Emflaza is medically necessary when the following criteria are met:
I. Initial Approval Criteria
A. Duchenne Muscular Dystrophy (must meet all):

1. Diagnosis of DMD confirmed by one of the following (a or b): a. Genetic testing (e.g., dystrophin deletion or duplication mutation found);
   b. If genetic studies are negative (i.e., no mutation identified), positive muscle biopsy (e.g., absence of dystrophin protein);
2. Prescribed by or in consultation with a neurologist;
   
   3. Age ≥ 2 years;
   4. Failure of a ≥ 6 month trial of prednisone, unless contraindicated or clinically significant adverse effects are experienced;
3. Documentation of member’s current weight in kg;
   
   6. Dose does not exceed 0.9 mg/kg per day.
      Approval duration: 6 months
      B. Other diagnoses/indications (must meet 1 or 2):

4. If this drug has recently (within the last 6 months) undergone a label change (e.g., newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, refer to one of the following policies (a or b): a. For drugs on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the no coverage criteria policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.33 for health insurance marketplace, and CP.PMN.255 for Medicaid; or Page 1 of 6

   CLINICAL POLICY Deflazacort b. For drugs NOT on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the non-formulary policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or

5. If the requested use (e.g., diagnosis, age, dosing regimen) is NOT specifically listed under section III (Diagnoses/Indications for which coverage is NOT authorized) AND criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business: CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid.
   II. Continued Therapy A. Duchenne Muscular Dystrophy (must meet all):
   
   1. Member meets one of the following (a or b): a. Currently receiving medication via Centene benefit or member has previously met initial approval criteria; b. Member is currently receiving medication and is enrolled in a state and product with continuity of care regulations (refer to state specific addendums for CC.PHARM.03A and CC.PHARM.03B);
   2. Member is responding positively to therapy;
   3. Documentation of member’s current weight in kg;
   4. If request is for a dose increase, new dose does not exceed 0.9 mg/kg per day. Approval duration: Medicaid/HIM – 12 months Commercial – 12 months or duration of request, whichever is less B. Other diagnoses/indications (must meet 1 or 2):
6. If this drug has recently (within the last 6 months) undergone a label change (e.g., newly approved indication, age expansion, new dosing regimen) that is not yet reflected in this policy, refer to one of the following policies (a or b): a. For drugs on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the no coverage criteria policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.33 for health insurance marketplace, and CP.PMN.255 for Medicaid; or b. For drugs NOT on the formulary (commercial, health insurance marketplace) or PDL (Medicaid), the non-formulary policy for the relevant line of business: CP.CPA.190 for commercial, HIM.PA.103 for health insurance marketplace, and CP.PMN.16 for Medicaid; or

7. If the requested use (e.g., diagnosis, age, dosing regimen) is NOT specifically listed under section III (Diagnoses/Indications for which coverage is NOT authorized) AND criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business: CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid.
   III. Diagnoses/Indications for which coverage is NOT authorized:
   A. Non-FDA approved indications, which are not addressed in this policy, unless there is sufficient documentation of efficacy and safety according to the off label use policies – Page 2 of 6

   CLINICAL POLICY Deflazacort CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid or evidence of coverage documents.
   IV. Appendices/General Information Appendix A: Abbreviation/Acronym Key DMD: Duchenne muscular dystrophy FDA: Food and Drug Administration Appendix B: Therapeutic Alternatives
   This table provides a listing of preferred alternative therapy recommended in the approval criteria. The drugs listed here may not be a formulary agent for all relevant lines of business and may require prior authorization.
   Dosing Regimen Drug Name Dose Limit/ Maximum Dose Varies based on weight prednisone 0.75 mg/kg/day PO (preferred) Alternative dosing regimens
   • 0.3 mg/kg/day PO (lesser efficacy and fewer adverse events) • 10 mg/kg/weekend PO Therapeutic alternatives are listed as Brand name® (generic) when the drug is available by brand name only and generic (Brand name®) when the drug is available by both brand and generic. Off-label Appendix C: Contraindications/Boxed Warnings • Contraindication(s): hypersensitivity to deflazacort or any of the inactive ingredients in Emflaza • Boxed warning(s): none reported Appendix D: General Information • Examples of positive response to corticosteroid therapy (e.g., Agamree, Emflaza, prednisone) include improvement in muscle strength tests (e.g., Medical Research Council [MRC] scale for muscle strength with 0 being no movement and 5 being normal strength), pulmonary function tests (e.g., forced vital capacity [FVC] and maximal expiratory pressure), walk tests (e.g., 6 minute walk test [6MWT] distance), and timed functional tests (e.g., standing from lying position; climbing 4 stairs; running/walking 30 feet; propelling a wheelchair 30 feet). In clinical trials, Emflaza has demonstrated similar efficacy to prednisone with regard to muscle strength, motor function, pulmonary function, and loss of ambulation. Emflaza may be associated with potentially less weight gain than prednisone; however, it may also be associated with more growth reduction and cataracts. In an evidence report published August 2019, the Institute for Clinical and Economic Review (ICER) concludes: “…we have moderate certainty that deflazacort has comparable or better net health benefits compared to prednisone.”
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   CLINICAL POLICY Deflazacort V. Dosage and Administration
   Indication DMD Dosing Regimen 0.9 mg/kg/dose PO QD VI. Product Availability
   • Tablets: 6 mg, 18 mg, 30 mg, 36 mg
   • Oral suspension: 22.75 mg/mL
   Maximum Dose 0.9 mg/kg/dose
   VII.