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.
Marc Blaustein never said it would be easy to develop a new drug for Duchenne muscular dystrophy. But even though he doesn't have the biggest organization, the most money or a full set of human data on key endpoints, he has been making some steady, early progress on his quest.
After a late-stage failure and a kiss-off from partner GlaxoSmithKline, Prosensa and its innovative treatment for Duchenne muscular dystrophy seemed to be on a downward slope. But now, after a series of "positive" meetings with the FDA, the company says it plans to submit its breakthrough-designated drug for approval this year, taking advantage of the agency's accelerated pathway.
Reversing an earlier rejection, European regulators are recommending early approval for PTC Therapeutics' ataluren, a treatment for Duchenne muscular dystrophy, news that more than doubled the biotech's share value in premarket trading.
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.
A class of drugs used to treat erectile dysfunction could also treat blood-flow problems related to Duchenne muscular dystrophy--a bright spot in a disease field marked by repeated clinical setbacks.
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.
After the markets closed last night, Prosensa issued an upbeat self-assessment of its 48-week extension data from a Phase II study of its lead drug drisapersen for Duchenne muscular dystrophy.
Patient groups are a potent force in the Duchenne muscular dystrophy field, where parents are known to take a vocal role in pushing regulators to help biotechs advance new therapies for the lethal genetic condition. Now one of those parents, J.P. Morgan vet Ilan Ganot, has reportedly been raising some serious cash to do deals on early-stage therapies that can be rushed through clinical development.