Pharmacy Prior Authorization Criteria

Emflaza (deflazacort) - PA, NF

Indications for Prior Authorization

Emflaza (deflazacort)

• For diagnosis of Duchenne muscular dystrophy (DMD)
  Indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

Criteria

Brand Emflaza, generic deflazacort

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of Duchenne muscular dystrophy (DMD)
AND
• Patient has received genetic testing for a mutation of the dystrophin gene [A, 2]
AND
• One of the following [A, 2]:
• Documentation of a confirmed mutation of the dystrophin gene
OR
• Muscle biopsy confirmed an absence of dystrophin protein
AND
• Patient is 2 years of age or older
AND
• Prescribed by or in consultation with a neurologist who has experience treating children
AND
• Dose will not exceed 0.9 milligrams per kilogram of body weight once daily
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 [B, 3-5]
AND
• One of the following:
• Trial and intolerance to generic deflazacort tablet (Applies to Brand Emflaza tablet only)
OR
• Trial and intolerance to generic deflazacort suspension (Applies to Brand Emflaza oral suspension only)

Brand Emflaza, generic deflazacort

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

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

Brand Emflaza, generic deflazacort tablet

Non Formulary

Length of Approval: 12 Month(s)

• Submission of medical records (e.g., chart notes, laboratory values) confirming diagnosis of Duchenne muscular dystrophy (DMD)
AND
• Patient has received genetic testing for a mutation of the dystrophin gene [A, 2]
AND
• Submission of medical records (e.g., chart notes, laboratory values) confirming one of the following [A, 2]:
• Documentation of a confirmed mutation of the dystrophin gene
OR
• Muscle biopsy confirmed an absence of dystrophin protein
AND
• Patient is 2 years of age or older
AND
• Prescribed by or in consultation with a neurologist who has experience treating children
AND
• Dose will not exceed 0.9 milligrams per kilogram of body weight once daily
AND
• Paid claims or submission of medical records (e.g., chart notes) confirming 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 [B, 3-5]
AND
• Both of the following (Applies to Brand Emflaza suspension only):
• Submission of medical records (e.g., chart notes) confirming the patient has experienced intolerance (e.g., allergy to excipient) to generic deflazacort suspension
AND
• Submission of medical records (e.g., chart notes) confirming generic deflazacort suspension has not been effective AND valid clinical rationale provided explaining how the Non-Formulary or Excluded Medication is expected to provide benefit when generic deflazacort suspension has not been shown to be effective despite having the same active ingredient

P & T Revisions

2024-11-11, 2024-07-08, 2024-06-11, 2024-04-03, 2023-05-04, 2022-05-05, 2021-08-10, 2021-04-22, 2020-04-09

References

1. Emflaza Prescribing Information. PTC Therapeutics, Inc. South Plainfield, NJ. June 2021.
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 4, 2024.

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End Notes

1. Genetic testing after a positive biopsy diagnosis of Duchenne muscular dystrophy (DMD) is mandatory [2]. However a muscle biopsy is not necessary if a positive genetic diagnosis is confirmed first. In rare cases, when a genetic test has been done but no mutation has been found, a muscle biopsy is the next necessary step for patients who have increased creatine kinase concentrations and symptoms consistent with DMD.
2. Prednisone 0.75 mg/kg/d should be considered the optimal prednisone dose in DMD. Over 12 months, prednisone 10 mg/kg/weekend is equally effective, although long term outcomes of this alternative regimens are unknown [3].

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Revision History

• 2024-11-11: Update to NF criteria
• 2024-07-08: Minor update to NF T/F section
• 2024-06-11: Addition of generic deflazacort suspension, update to PA & NF T/F requirement
• 2024-04-03: Annual review: Added generic deflazacort tablet formulation to guideline and updated criteria (includes formulary strategy). Background/formatting updates.
• 2023-05-04: Annual review: Updated Non Formulary criteria and background.
• 2022-05-05: Annual review: Background updates.
• 2021-08-10: Updated guideline name and guideline type, and added EHB formulary.
• 2021-04-22: Annual Review
• 2020-04-09: Annual Review

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