Aim: Eteplirsen, golodirsen and casimersen are phosphorodiamidate morpholino oligomers (PMOs) that have received, based on biomarker data, accelerated approval from the US FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients with pathogenic variants amenable to 51, 53 and 45 exon skipping, respectively. The objectives of this study were to describe patient demographic and baseline functional characteristics, safety and treatment continuation in patients with DMD who were treated with a commercially available PMO in the US from the ongoing phase IV, multicenter, prospective, observational EVOLVE study. Patients 23 received golodirsen, mean (SD) age 13.3 (4.25) years; and 12 were treated with casimersen, mean (SD) age 16.1 (7.21) years. Mean (SD) total duration of treatment was 6.2 (1.92) years for eteplirsen, 2.4 (0.83) years for golodirsen and 1.7 (0.62) years for casimersen. All PMOs demonstrated favorable safety profiles, with no treatment-emergent serious adverse events related to treatment. Most patients taking eteplirsen (95.2%, n = 120) continued treatment. Among the 85 patients who were ambulatory at treatment initiation, 37 patients lost ambulation, 34 (91.9%) of whom remained on eteplirsen. Conclusion: Consistent with the safety findings from previous clinical trials, eteplirsen, golodirsen and casimersen showed favorable safety profiles in patients with DMD in routine clinical practice. EVOLVE will continue to describe long-term clinical outcomes Clinical Trial Registration Number: NCT06606340.
Tian et al. (Fri,) studied this question.
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