Sarepta Therapeutics announces that their drug; Eteplirsen has
shown some amazing results.
Participants in the trial have been shown to be producing
dystrophin and achieving clinically significant benefits as
measured by the 6-Minute Walk Test, this is after 48 Weeks of
The results are from the Phase IIb Study in Duchenne Muscular
Oct 03, 2012 Sarepta Therapeutic, announced that treatment with its
lead exon-skipping compound, eteplirsen, met its primary efficacy
endpoint. This means that it an increase in novel dystrophin has
been shown and participants in the trial achieved a significant
clinical benefit on the primary clinical outcome, measured by the
6-minute walk test (6MWT) compared with the placebo/delayed
treatment cohort .
Eteplirsen administered once weekly at either 30 mg/kg or 50 mg/kg
for 48 weeks (n=8) resulted in a statistically significant increase
(p≤0.001) in dystrophin-positive fibers to 47.0% of
The placebo/delayed treatment cohort, which had received 24 weeks
of eteplirsen at either 30 mg/kg or 50 mg/kg following 24 weeks of
placebo (n=4), also showed a statistically significant increase in
dystrophin-positive fibers to 38.3% of normal (p≤0.009).
"These data represent a significant milestone and a
defining moment of progress and hope for patients with DMD and
their families, as well as for those of us in the scientific
community who have been pursuing potential treatments for this
devastating and deadly disease for decades," said Jerry Mendell,
M.D., Director of the Centers for Gene Therapy and Muscular
Dystrophy at Nationwide Children's Hospital and principal
investigator of the Phase IIb study.
Dr. Mendell added, "By addressing the underlying cause of
DMD, eteplirsen has demonstrated unparalleled effects on enabling
dystrophin production and slowing the progression of the disease as
measured by the 6-minute walk test, with no treatment associated
While eteplirsen is targeted to DMD patients with a
specific genetic mutation, I think the implications for all DMD
patients with related genetic mutations are clearly
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