Santhera reported positive topline results from an analysis of long-term data, including first assessments from the ongoing, open-label, multicenter GUARDIAN study evaluating AGAMREE (vamorolone) in patients with Duchenne muscular dystrophy (DMD). >>> What is Agamree?
Featured Results
- Comparable long-term effectiveness to standard of care corticosteroids over 5 years
- Normal growth maintained, in contrast to growth suppression commonly seen with standard of care corticosteroids
- Significantly lower rate of vertebral fractures reported with AGAMREE
- Lower incidence of cataracts than normally seen for DMD patients on corticosteroids
110 patients were analyzed
The long-term analysis included corticosteroid-naïve patients who began AGAMREE treatment between four and seven years of age in clinical studies and continued through various access programs, including the GUARDIAN study. Data from up to 110 patients were analyzed, with patient numbers varying by analysis depending on data availability. In this long-term analysis, patients had received AGAMREE for up to eight years, with a median follow-up of about five years. Most patients remained on higher doses (4–6 mg/kg/day) in real-world clinical settings over the observation period.
Patients treated with AGAMREE maintained motor function over extended follow-up, demonstrating durable efficacy as measured by time to loss of ambulation comparable to standard of care corticosteroids (p=0.91). In pre-specified subgroup analyses, no differences were observed compared with either daily deflazacort or prednisone.
Safety and tolerability
Importantly, the data continue to support a differentiated safety and tolerability profile versus traditional corticosteroids. Patients treated with AGAMREE experienced a significantly lower rate of vertebral fractures (p=0.0061), maintained normal growth without the stunting seen with standard of care corticosteroids (p<0.0001), and showed fewer cases of cataracts than those treated with glucocorticoids, including a notably lower incidence versus deflazacort (p<0.015). Additionally, to date, no cases of glaucoma were observed. On average, changes in BMI or weight, when matched for height, did not differ, and no new safety signals were observed.
Detailed results are being submitted for presentation at a major international scientific conference in Q1 2026. In line with standard scientific practice, the complete data will be shared publicly following the conference presentation, once the organizers have completed their review and presentation process. In addition, further readouts from the GUARDIAN study are planned over the next three years, focusing on a broader set of efficacy and safety outcomes, including assessments of upper limb function, pubertal development, eye health, and cardiac function, as well as other relevant parameters.
Follow This Page >>> All Clinical Trials for Duchenne
