Delandistrogene moxeparvovec, also known as Elevidys, is an adeno-associated virus vector-based therapy that has been approved for patients aged 4 years and older with Duchenne muscular dystrophy (DMD), receiving its initial FDA approval in June 2023 and an expanded approval in June 2024.

Recent findings from the EMBARK study, presented at the ASGCT Annual Meeting, highlighted significant clinical benefits of this therapy in 8- and 9-year-old patients with DMD.

The ongoing EMBARK trial, which includes 63 participants who received the gene therapy, reported meaningful benefits over a two-year period when compared to an external control group, particularly in North Star Ambulatory Assessment (NSAA) scores and functional tests.

Results from the trial demonstrated a notable 2.88-point increase in NSAA scores and a 2.06-second improvement in rising from the floor over two years, confirming the therapy's efficacy.

One year after treatment, patients showed a significant 4.75-point increase in NSAA scores, a 6.87-second improvement in time to rise from the floor, and a 4.76-second enhancement in the 10-meter walk/run test, all statistically significant.

The study involved 14 patients who transitioned from a placebo group and exhibited notable functional improvements in motor skills after receiving the gene therapy.

Louise Rodino-Klapac, chief scientific officer at Sarepta Therapeutics, emphasized the importance of these results, indicating that gene therapy can significantly slow functional decline in patients at a critical age.

Safety analysis from the trial revealed no new safety signals, with 15 patients experiencing treatment-related adverse events, and only one case of serious adverse effects that resolved without complications.