Positive Impact of Ataluren on Walking Ability in Duchenne Muscular Dystrophy: Jeffrey M. Statland, MD
At the 2023 AAN Annual Meeting, the neuromuscular disease specialist and professor of neurology at the University of Kansas Medical Center talked about the phase 3 study investigating ataluren for Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]
WATCH TIME: 5 minutes
“There have been several clinical trials of ataluren in Duchenne muscular dystrophy, including a prior phase 2 and phase 3 study. We've learned a lot from the data in those studies, including about this new therapeutic that may be important for these boys. But also, we learned a lot about outcome measures designing the clinical trials.”
Duchenne muscular dystrophy (DMD), a severe neuromuscular disorder, is caused by a lack of functional dystrophin. Ataluren (Translarna; PTC Therapeutics), designed as a treatment for patients with nonsense mutation (nm) DMD, as it promotes readthrough of an in-frame premature stop codon to produce full-length dystrophin. Results from the phase 3 Study 041 (NCT03179631) of ataluren further confirmed the therapy’s positive risk-benefit profile over a 72-week period in patients with DMD and a nonsense mutation.1
The results were recently presented by Jeffrey Statland, MD, at the 2023 American Academy of Neurology (AAN) Annual Meeting, April 22-27, in Boston, Massachusetts. The analysis, which comprised of 95 patients (51.9%) on ataluren and 67 patients (38.1%) on placebo group, showed that the median time to 10% persistent worsening was 74.3 weeks (95% CI, 59.1-NA) for the ataluren group compared with 48.0 weeks (95% CI, 36.0-60.9) in the placebo group (log-rank P = .0078), resulting in an HR of 0.7 (95% CI, 0.5-0.9).
There were significant differences compared with placebo, observed in mean 6-minute walk distance (6MWD) in both the intent-to-treat (ITT) population (ataluren, n = 183; placebo, n = 176) and the subgroup of boys with 300 m to 400 m 6MWD (ataluren, n = 86; placebo, n = 83). The change from baseline and rate of change supported ataluren in the ITT population (14.4 m; 0.20 m per week; P = .0248) and 300 m to 400 m 6MWD subgroup (24.2 m; 0.34 m per week; P = .0310).
Statland, a professor of neurology at the University of Kansas Medical Center in Kansas City, Kansas, sat down with NeurologyLive® in an interview at the meeting to discuss the objective of the phase 3 study. He also talked about the logistical challenges the study overcame involving participant recruitment during the COVID-19 pandemic, as well as key findings regarding the effect of ataluren on walking ability in boys with DMD.
Click here for more coverage on AAN 2023.
