Drug updated on 9/4/2024
Dosage Form | Tablet (oral; 6 mg, 18 mg, 30 mg, 36 mg); Suspension (oral; 22.75 mg/ml) |
Drug Class | Corticosteroids |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- • Indicated to treat Duchenne muscular dystrophy in patients ≥ 2 years of age.
Latest News
Summary
- Emflaza (deflazacort) is indicated to treat Duchenne muscular dystrophy in patients ≥ 2 years of age.
- This summary is based on the review of two systematic review(s)/meta-analysis(es). [1-2]
- Deflazacort demonstrated a significant improvement in a 6-minute walk distance with a least-squares mean difference of 39.54 meters (95% CI: 13.799, 65.286; p = 0.0026) and a lower decline of 28.3 meters (95% CI: 5.7, 50.9) compared to prednisone/prednisolone in ambulatory boys with nonsense mutation Duchenne muscular dystrophy (DMD).
- Deflazacort-treated patients experienced lower declines in timed function tests, including 2.9 seconds on rise from supine (95% CI: 0.9, 4.9 seconds) and 2.3 seconds on the 4-stair climb (95% CI: 0.5, 4.1 seconds), compared to prednisone/prednisolone.
- Deflazacort led to a lower decline of 2.9 points on the North Star Ambulatory Assessment linearized score (95% CI: 0.1, 5.8) in ambulatory boys with nonsense mutation DMD.
- No safety information was found in the reviewed studies.
- The studies focused on ambulatory boys with Duchenne muscular dystrophy (DMD) with a nonsense mutation, prior corticosteroid use, and stable baseline dosing; findings indicated that deflazacort was more effective in delaying the loss of physical milestones compared to prednisone/prednisolone in this specific population.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Emflaza (deflazacort) Prescribing Information. | 2021 | PTC Therapeutics, Inc., South Plainfield, NJ |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy. | 2021 | Journal of Comparative Effectiveness Research |
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: a meta-analysis of disease progression rates in recent multicenter clinical trials. | 2020 | Muscle and Nerve |
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Clinical practice guidelines for the diagnosis and management of Duchenne muscular dystrophy: a scoping review. | 2024 | Frontiers in Medicine |
Management of adrenal insufficiency risk after long-term systemic glucocorticoid therapy in Duchenne muscular dystrophy: Clinical Practice Recommendations. | 2019 | Journal of Neuromuscular Diseases |