EMFLAZA (deflazacort)

Emflaza (deflazacort)

SELF ADMINISTRATION - ORAL

Indications for Prior Authorization:

• Treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older

Coverage Criteria:

For diagnosis of Duchenne muscular dystrophy (DMD):

• Dose does not exceed 0.9 milligrams per kilogram of body weight once daily, AND
• Diagnosis of Duchenne muscular dystrophy (DMD), AND
• Patient is 2 years of age or older, AND
• Prescribed by or in consultation with a neurologist who has experience treating children, AND
• Patient has received genetic testing for a mutation of the dystrophin gene, AND
• One of the following:
  • Documentation of a confirmed mutation of the dystrophin gene, or
  •  Muscle biopsy confirmed an absence of dystrophin protein, AND
• Patient has had a trial and failure or intolerance to prednisone or prednisolone given at a dose of 0.75 mg/kg/day or 10 mg/kg/weekend, AND
• One of the following:
  • Trial and intolerance (e.g., allergy to excipient) to generic deflazacort tablet (Applies to Brand Emflaza tablet only)
  • Trial and intolerance (e.g. allergy to excipient) to generic deflazacort oral suspension (Applies to Brand Emflaza suspension only)
     

Reauthorization Criteria:

For diagnosis of Duchenne muscular dystrophy (DMD):

• Dose does not exceed 0.9 milligrams per kilogram of body weight once daily, AND
• Patient has experienced a benefit from therapy (e.g., improvement or preservation of muscle strength), AND
• One of the following:
  • Trial and intolerance (e.g., allergy to excipient) to generic deflazacort tablet (Applies to Brand Emflaza tablet only)
  • Trial and intolerance (e.g. allergy to excipient) to generic deflazacort oral suspension (Applies to Brand Emflaza suspension only)

Coverage Duration: 

• Initial: 1 year
• Reauthorization: 1 year

Additional Information: 

• Emflaza Tablets: can be administered whole or crushed and taken immediately after mixing with applesauce.
• Emflaza Oral Suspension: Use only the oral dispenser provided with the product.  After withdrawing the appropriate dose into the oral dispenser, slowly add the Emflaza Oral Suspension into 3 to 4 ounces of juice (except grapefruit juice) or milk and mix well. The dose should then be administered immediately. Discard any unused Emflaza Oral Suspension remaining after 1 month of first opening the bottle.
• Dosing:
  • Approximately 0.9mg/kg/day once daily (round up to nearest possible dose when using tablets, or round up to nearest tenth of a mL when using suspension)
  • Concomitant moderate of strong CYP3A4 inhibitors (e.g., clarithromycin, fluconazole, diltiazem, verapamil): reduce dose to 1/3 of usual dose
  • Avoid use with moderate or strong CYP3A4 inducers

Policy Updates:

• 5/1/2017 – New policy approved by P&T
• 11/14/2023 – Updated age requirement, added additional criteria for initial review, added reauthorization criteria
• 09/01/2024 – Addition of generic deflazacort tablets step through for Brand Emflaza tablets and suspension (P&T 08/20/2024) (P&T Meeting August)

References:

1. Emflaza Prescribing Information. PTC Therapeutics, Inc. South Plainfield, NJ. Feb 2023. 
2. Bushby K, Finkel R, Birnkrant DJ, et al; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010;9(1):77-93.
3. Gloss D, Moxley RT 3rd, 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):465-72.
4. Griggs RC, Miller JP, Greenberg CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016 Nov 15;87(20):2123-2131. 
5. FDA Center for Drug Evaluation and Research. Medical Review [Application Number 208684Orig1s000, 208685Orig1s000]. FDA Web site. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/208684,208685Orig1s000MedR.pdf. Accessed March 30, 2023.