In an uncommon move, the FDA has publicly weighed in on an in-development drug, clarifying the status of Sarepta Therapeutics' closely watched Duchenne muscular dystrophy treatment eteplirsen in light of an earlier setback that tanked the biotech's shares.
After a behind-the-scenes squabble spilled out into the public, Sarepta has replaced the chairman of its board and reaffirmed its support of CEO Chris Garabedian, looking to move on from an embarrassing distraction and focus on its lead drug program.
The FDA's Janet Woodcock may be Sarepta CEO Chris Garabedian's favorite person today.
After Sarepta Therapeutics' former head scientist blamed his swift ouster on "serious disagreements" with CEO Chris Garabedian, the company has moved to limit its chief executive's power, according to a report, especially when it comes to meetings with the FDA over the biotech's much-scrutinized lead drug.
Sarepta Therapeutics' terse dismissal of its top scientist on Thursday has led to a swirl of rumors and anonymous-sourced reports of boardroom clashes, incompatible personalities and a possible coup attempt, bringing to light worrisome details on a company already under the microscope.
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.
GlaxoSmithKline and Prosensa today conceded defeat in the Phase III study for drisapersen, one of two closely-watched therapies for Duchenne muscular dystrophy which had been vying for the lead in the field. Investigators reported that the therapy failed to significantly improve walking distance in patients.