The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed unmet needs that remain, chiefly concerning widening the criteria for therapies and trials.
“There's still significant unmet need. While the gene therapy approval is thrilling for all of us, there's still a narrow window of 4- to 5- year-olds. So we have many people younger than 4 and many people over the age of 5, that are still in need of treatment. So, I think while the landscape is really amazing, and building every single day... time matters to these people.Time means progression of disease.”
The FDA approved the first gene therapy in the muscular dystrophy field, delandistrogene moxeparvovec (Elevidys) for the treatment of Duchenne muscular dystrophy (DMD), in 2023, but many questions remain to be answered and data remain to be seen. To this end, Sarepta Therapeutics recently announced data from the global, pivotal, phase 3 EMBARK study (Study SRP-9001-301; NCT05096221) that showed that the study had failed its primary end point.
EMBARK failed to reach statistical significance in its key primary end point of change in North Star Ambulatory Assessment (NSAA) scores at week 52 compared to placebo. However, change in time to rise (TTR) and 10-meter walk test (10MWT), 2 key secondary end points, were statistically significant across all age groups compared to placebo.
CGTLive spoke with Pat Furlong, BSN, RN, founding president and chief executive officer, Parent Project Muscular Dystrophy (PPMD), to learn more about her thoughts on the changing treatment landscape for DMD. She discussed unmet research and treatment needs that remain, including investigating therapies for a wider range of people with DMD, people with neutralizing antibodies against adeno-associated virus vectors, and eventually, people that may need to be redosed with gene therapy.