Following promising results in slowing heart function decline in patients with DMD, Eteplirsen (Exondys 51) continues to show potential in addressing key aspects of the disease. The latest data from the phase 4 EVOLVE study presented at the 2025 MDA Conference revealed that eteplirsen significantly delayed loss of ambulation (LOA), providing further evidence of the drug’s ability to impact the course of DMD in a clinical setting.
What is Eteplirsen EVOLVE study?
The EVOLVE study, designed to assess eteplirsen’s effects on LOA in routine clinical practice, included 33 eteplirsen-treated patients and 75 external controls from 5 major studies. After adjusting for baseline differences through inverse probability treatment weighting (IPTW), the study reported that the risk of LOA was reduced by 62% in eteplirsen-treated patients. Additionally, the median age at LOA was significantly higher for treated patients (15.3 years) compared with external controls (11.3 years), further underscoring the treatment’s effectiveness. [Eteplirsen (Exondys 51)]
Result of EVOLVE study
LOA was determined by patient-reported wheelchair use, North Star Ambulatory Assessment (NSAA) scores, and a 10-meter walk/run (10MWR) score of at least 30, validated by the attending physician. Eteplirsen-treated patients experienced a median age of 15.3 years at LOA, significantly later than the 11.3 years in the control group, indicating a delay in disease progression.
