Sarepta's muscular dystrophy therapy fails to meet main goal in
late-stage trial
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[October 31, 2023]
(Reuters) -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.
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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.
(Reporting by Pratik Jain in Bengaluru; Editing by Krishna Chandra
Eluri and Shailesh Kuber)
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