Drug updated on 10/30/2024
Dosage Form | Tablets (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 for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.
Latest News
Summary
- This summary is based on the review of six systematic review(s)/meta-analysis(es). [1-6]
- Cardiac Function in 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 TTSTANDV, TTRWV, 6MWT, and 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 Title | Year | Source |
---|---|---|
Emflaza (deflazacort) Prescribing Information. | 2024 | PTC Therapeutics, Inc., Warren, NJ |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Predictors of cardiac disease in duchenne muscular dystrophy: a systematic review and evidence grading | 2024 | Orphanet Journal of Rare Diseases |
Efficacy and safety of different doses of vamorolone in boys with Duchenne muscular dystrophy: a systematic review and network meta-analysis | 2024 | Frontiers in Neurology |
Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis | 2024 | Journal of Neuromuscular Diseases |
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy | 2021 | Journal of Comparative Effectiveness Research |
The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy | 2020 | Journal of Managed Care & Specialty Pharmacy |
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials | 2020 | Muscle & 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 | 2020 | Journal of Neuromuscular Diseases |