Deflazacort

(Emflaza®)

Emflaza®

Drug updated on 5/17/2024

Dosage FormTablet (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 to treat Duchenne muscular dystrophy in patients ≥ 2 years of age.

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Summary
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  • Deflazacort (Emflaza) is indicated for treating Duchenne muscular dystrophy in patients aged 2 years and above. It has been shown to significantly improve the 6-minute walk distance with a least-squares mean difference of 39.54 meters compared to prednisone/prednisolone, indicating a clinically meaningful delay in the loss of physical milestones over 48 weeks.
  • This information was derived from two systematic reviews/meta-analyses that focused on Emflaza's comparison with prednisone/prednisolone for treating Duchenne Muscular Dystrophy.
  • Both studies primarily investigated ambulatory boys with Duchenne Muscular Dystrophy who had at least a six-month history of prior corticosteroid use and stable dosing, emphasizing early intervention as standard care for delaying disease progression.
  • In addition to improving walking distance, patients treated with deflazacort also showed significant improvements in their ability to climb and descend stairs.
  • Although safety data directly comparing deflazacort vs. prednisone/prednisolone were not provided within these studies, slower rates of functional decline suggest a potentially favorable risk-benefit ratio for deflazacort among this patient population, considering the long-term usage implications due to its chronic nature of therapy management.
  • Overall effectiveness indicates that deflazacort is more efficacious in slowing down the progression of Duchenne muscular dystrophy when compared against prednisone or prednisolone, based on lesser declines observed across various physical functions such as stair climbing capabilities and overall ambulatory assessments.