After months of back and forth with regulators yoked its share price up and down, Sarepta Therapeutics is all-in on its odds of winning FDA approval for its Duchenne muscular dystrophy treatment, putting up $25 million for a manufacturing plant to produce the drug.
After keeping investors on a roller coaster ride of speculation for months, Sarepta says it now plans to shoot for an accelerated approval of its Duchenne muscular dystrophy drug eteplirsen. The biotech says it will file for an early approval by the end of this year after launching a slate of new clinical trials to get the data that the FDA is looking for.
Sarepta's up-and-down quest to get eteplirsen in the hands of patients with Duchenne muscular dystrophy is looking a little sunnier thanks to more promising data and an optimistic reading of some FDA tea leaves, news that sent the biotech's shares up 35% on Thursday.
In what has been one of the most hotly contested issues among biotech investors this year, many had bet big that the FDA would push through an accelerated approval for eteplirsen based on some very promising results from a mid-stage study. The study, though, only included a dozen boys, making the prospect of an early approval problematic at best.
Drisapersen and eteplirsen have been angling to become the first approved therapy for lethal, muscle-wasting cases of Duchenne muscular dystrophy. So when investigators pulled back the covers from a new block of data on GlaxoSmithKline's drisapersen, the analysts immediately went to work to see how it stacked up against Sarepta's eteplirsen.
Shares of Sarepta were boosted this morning after the biotech announced that it reaped another round of promising results from a closely-watched Phase IIb study of its experimental treatment for Duchenne muscular dystrophy.
Sarepta Therapeutics has kept hopes alive for gaining accelerated approval for its lead drug eteplirsen for Duchenne muscular dystrophy.
GlaxoSmithKline investigator John Kraus reviewed the long-awaited mid-stage results for its Duchenne muscular dystrophy drug drisapersen this afternoon, revealing that boys taking the experimental therapy benefited with a significant improvement in walking distance compared to a placebo arm.
The clock is ticking for Sarepta, a multitude of biotech investors and the boys who suffer from Duchenne muscular dystrophy.
Sarepta Therapeutics has seen demand for its stock spike since the company revealed impressive long-term data two months ago from a mid-stage study of its therapy eteplirsen in patients with Duchenne muscular dystrophy.