Deflazacort

(Emflaza®)

Emflaza®

Drug updated on 12/11/2024

Dosage FormTablets (oral; 6 mg, 18 mg, 30 mg, 36 mg); Suspension (oral; 22.75 mg/ml)
Drug ClassCorticosteroids
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

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

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Summary
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  • This summary is based on the review of six systematic review(s)/meta-analysis(es). [1-6]
  • Cardiac Function in duchenne muscular dystrophy (DMD): High-quality evidence shows that deflazacort is associated with preserved left ventricular ejection fraction (LVEF) and improved fractional shortening (FS). Low-quality evidence suggests deflazacort, prednisone, and prednisolone are linked to improved ejection fraction (EF) and reduced risks of cardiomyopathy, ventricular dysfunction, and heart failure-related mortality.
  • Motor Function in DMD: Deflazacort provides superior motor function outcomes compared to prednisone/prednisolone, with significant improvements in 6-minute walk distance (6MWD), 4-stair climb, and 4-stair descend over 48 weeks. Vamorolone 6 mg/kg/day improved motor function compared to the 2 mg/kg/day dose, with notable improvements in time to stand from supine velocity (TTSTANDV), time to run/walk 10 m velocity (TTRWV), 6-min walk test (6MWT), and time to climb 4 stairs velocity (TTCLIMBV).
  • There is no safety information available in the reviewed studies.
  • There is no population types or subgroups information available in the reviewed studies.

Product Monograph / Prescribing Information

Document TitleYearSource
Emflaza (deflazacort) Prescribing Information.2024PTC Therapeutics, Inc., Warren, NJ

Systematic Reviews / Meta-Analyses

Clinical Practice Guidelines