In a recent late-stage clinical trial, Sarepta Therapeutics’ gene therapy designed to treat Duchenne muscular dystrophy (DMD), a progressive muscle-wasting genetic disorder, failed to achieve its primary goal when administered to patients aged 4 to 7 years. Specifically, the therapy did not demonstrate statistical significance in the total assessment score of motor function when compared to patients who received a placebo over a 52-week period. DMD primarily affects young boys and typically results in a limited life expectancy beyond one’s thirties.

The gene therapy, known as Elevidys, had previously received accelerated approval from the U.S. health regulator for children aged 4 to 5 years who can walk, as opposed to the initial application seeking approval for all walking DMD patients. The company highlighted that Elevidys did meet all secondary study objectives with statistically significant outcomes and did not show any new safety concerns.

Sarepta plans to request an update from the U.S. Food and Drug Administration (FDA) to expand the therapy’s usage to other age groups based on the trial findings. Elevidys is a one-time treatment, representing a potential shift in the treatment paradigm for DMD patients, as current therapies necessitate ongoing use. Of the four “exon-skipping” therapies intended for a specific subset of DMD patients with specific genetic mutations, three are developed by Sarepta. These therapies require weekly infusions and function by skipping specific gene segments, known as exons, in order to promote the formation of shorter dystrophin protein forms.

Roche holds commercial rights to Elevidys outside the United States, while Sarepta retains responsibility for clinical development and shares the associated costs with Roche.

The disappointing results from Sarepta Therapeutics’ trial have led to a significant drop in the company’s stock value. On the other hand, it raised questions about how regulators handle gene therapies with unclear outcomes but high medical needs.

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