Five-year data of AGAMREE® (vamorolone) in patients with DMD show improved safety profile with comparable effectiveness to standard of care corticosteroids

Core Insights - Santhera Pharmaceuticals reported positive topline results from the long-term analysis of the GUARDIAN study evaluating AGAMREE® (vamorolone) for Duchenne muscular dystrophy (DMD) patients [1][5] Long-term Analysis - The analysis included corticosteroid-naïve patients aged four to seven who received AGAMREE for up to eight years, with a median follow-up of about five years [2] - Up to 110 patients were analyzed, with most remaining on higher doses (4–6 mg/kg/day) during the observation period [2] Efficacy - Patients treated with AGAMREE maintained motor function over extended follow-up, showing durable efficacy comparable to standard corticosteroids in terms of time to loss of ambulation (p=0.91) [3] - No significant differences were observed in pre-specified subgroup analyses compared to daily deflazacort or prednisone [3] Safety and Tolerability - AGAMREE demonstrated a differentiated safety profile, with a significantly lower rate of vertebral fractures (p=0.0061) and normal growth without stunting, unlike standard corticosteroids (p<0.0001) [4] - Fewer cases of cataracts were reported compared to glucocorticoids, including a notably lower incidence versus deflazacort (p<0.015) [4] - No cases of glaucoma were observed, and changes in BMI or weight did not differ when matched for height [4] Future Developments - Detailed results will be presented at a major international scientific conference in Q1 2026, with complete data to be shared publicly afterward [5] - Further readouts from the GUARDIAN study are planned over the next three years, focusing on a broader set of efficacy and safety outcomes [5] Expert Commentary - Experts in the field expressed encouragement regarding the long-term efficacy and safety profile of AGAMREE, highlighting its potential benefits over conventional corticosteroids [7][9]