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Sarepta’s muscular dystrophy therapy fails to meet main goal in late-stage trial

In this photo illustration a Sarepta Therapeutics logo seen displayed on a smartphone with the stock market graphic in the background.

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Sarepta Therapeutics‘ gene therapy to treat Duchenne muscular dystrophy (DMD), a progressive muscle-wasting disorder, failed to meet the main goal of a late-stage trial when tested in patients between 4 and 7 years, the company said on Monday.

The therapy failed to meet statistical significance in total assessment score from a measure to test motor function when compared to placebo-treated patients at 52 weeks.

The gene therapy is the first of its kind for DMD, an inherited disorder, that almost always affects young boys. Its patients rarely survive beyond their thirties.

The therapy, branded as Elevidys, was granted accelerated approval by the U.S. health regulator in June for children aged between 4 and 5 years who can walk, versus the company’s initial application seeking approval for all DMD patients who can walk.

The company said the therapy met all secondary study goals with statistically significant results and no new safety signals were observed.

Sarepta said it plans to seek the U.S. Food and Drug Administration’s update for the expanded use of the therapy in other age groups based on the trial results.

Elevidys, a one-time treatment, is expected to change the way DMD patients are treated as current therapies require regular use.

Current options for DMD patients are limited. Of four new “exon-skipping” therapies – intended for a smaller group of patients with specific genetic mutations – three are from Sarepta.

They require weekly infusions and work by skipping specific parts of genes, called exons, with the aim of allowing the body to form shorter forms of the dystrophin protein

Roche acquired the commercial rights to Elevidys outside the United States under a 2019 deal. Sarepta remains in charge of clinical development, but it splits the costs with Roche.

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