US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

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RE: FDA Says That New Drug Application Filing for Eteplirsen is Premature


Sarepta Therapeutics Inc. has received word from the U.S. Food and Drug Administration (FDA) that the agency does not consider experimental exon-skipping drug eteplirsen for Duchenne muscular dystrophy (DMD) a good candidate for an accelerated approval pathway at this time. The FDA is concerned that the functional data (six-minute walk test) and the dystrophin protein data from the phase 2b trial of eteplirsen do not provide sufficient evidence of effectiveness of the drug. Sarepta says it remains committed to eteplirsen and will work with the FDA on a possible redesign of the upcoming, confirmatory trial of eteplirsen, while at the same time continuing to explore with the agency the possibility of moving the drug through an accelerated approval pathway.


The following quote may be attributed to Jane Larkindale, Ph.D., MDA vice president for research.

"We share the community's disappointment that there will be a probable delay in filing for approval of eteplirsen and in starting the confirmatory trial," said Jane Larkindale, MDA's vice president for research. "However, we want families to understand that the FDA is not saying that eteplirsen doesn't work. The agency is saying it wants to make sure that we are measuring any effects of eteplirsen accurately — which is important not just for this drug but for all drugs to come.”

"We need to ensure we neither underestimate nor overestimate the effects of a new treatment," Larkindale said, noting that the FDA's decision reflects this important point. "On behalf of the families we serve, MDA is committed to finding ways to measure treatment effects accurately, and to advancing safe and effective lifesaving drugs as quickly as possible."