Sarepta and Roche’s confirmatory Phase III EMBARK study for Duchenne muscular dystrophy gene therapy, Elevidys, failed to meet its primary endpoint, complicating plans for broader approval. Although patients receiving Elevidys showed a more significant improvement in their functional mobility (2.6-point) than the placebo group (1.9-point), the difference was not statistically significant (p-value of 0.24). However, secondary endpoints related to standing and walking abilities were statistically significant. The FDA had granted Elevidys accelerated approval for a limited group of boys aged 4 and 5. The industry closely watched the trial, hoping Elevidys could be approved for older patients. Sarepta intends to request an update to expand the treatment’s indication to all patients despite the missed primary endpoint. A final decision regarding this update and possible broad label lies with FDA regulators.
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[Source: Endpoints, October 30th, 2023]