Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD
Publication: Journal of Comparative Effectiveness Research
Abstract
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 & methods: Patients who received or initiated treatment with a PMO at the time of study enrollment as prescribed by treating physicians as part of routine care were included. Approximately 300 patients will be enrolled across the three PMOs. Results: As of this 2023 interim data report, 161 patients were enrolled: 126 were treated with eteplirsen (enrollment complete), mean (standard deviation [SD]) age 14.0 (5.51) years; 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
Plain language summary: assessing real-world experience with eteplirsen, golodirsen and casimersen
What is this article about?
Duchenne muscular dystrophy (DMD) is a rare genetic disease in which there is a lack of a muscle protein called dystrophin. Because dystrophin protein is needed for muscle strength and function, over time, people lose the ability to walk and develop breathing and heart problems. In this ongoing real-world study named EVOLVE, investigators collected data from individuals with DMD who were receiving eteplirsen, golodirsen or casimersen from routine clinical visits. EVOLVE is the first and largest study to gather information on the safety and effectiveness of these approved therapies in individuals with DMD. This article includes safety results.
What were the results?
This interim data report included 126 people receiving eteplirsen, 23 receiving golodirsen and 12 receiving casimersen. The results of this study mainly focused on eteplirsen usage, with individuals receiving treatment for 6.2 years at an average age of 14 years. There were no new safety concerns, and most individuals continued using eteplirsen while in the study.
What do the results mean?
The safety results of eteplirsen in this real-world study are similar to those of previous studies of eteplirsen. Collection of data at the start of a study helps investigators understand the types of individuals who receive these medicines in a real-world setting. It is valuable for individuals with DMD to continue completing tests that measure breathing and heart function so that researchers can evaluate how these medicines may change these important functions.
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Received: 28 June 2025
Accepted: 9 February 2026
Published online: 10 April 2026
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Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD. (2026) Journal of Comparative Effectiveness Research. DOI: 10.57264/cer-2025-0108
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