|Sarepta CEO Chris Garabedian|
The FDA's Janet Woodcock may be Sarepta CEO Chris Garabedian's favorite person today.
With the news about the company focused this week on the embarrassing move by Sarepta's ($SRPT) board to curtail Garabedian's direct involvement with the FDA after some rumored run-ins with regulators, Woodcock issued a letter to an influential patient group in the Duchenne muscular dystrophy field that committed the agency to "explore the use of all potential pathways for the approval of drugs for Duchenne muscular dystrophy, including accelerated approval, as appropriate." Sarepta was also cited by name in the letter.
So yesterday Sarepta's shares climbed north as investors focused in on the positive implications for the company, where the CEO has been insisting that the agency has turned supportive of its effort to get an early OK for eteplirsen.
There isn't actually anything all that new, or really all that noteworthy, about the statement from Woodcock, an influential insider at the agency. The FDA isn't in the business of publicly opposing new classes of drugs, especially when there are no therapies for a disease like DMD. Patients, and particularly the parents of the young boys afflicted by this lethal muscle-wasting disease, are demanding action now. And anything less than leaving the door open to an early OK would have likely incited a riot.
|CDER Director Janet Woodcock|
But this time might be different. There's reason to believe that the agency has really changed its attitude about accelerating approvals for DMD drugs, and Woodcock may be open to much more than placating angry parents.
The open-door policy being pronounced now comes among some clear signs that regulators on both sides of the Atlantic have been changing their stance to the three experimental therapies now racing for an early approval. Following a setback at the FDA, Sarepta led the pack, executing a quick u-turn with its plan for an accelerated approval after meeting with regulators. The biotech was quickly followed up by Prosensa ($RNA), which is advancing drisapersen with some renewed confidence after a stinging Phase III setback. PTC Therapeutics ($PTCT) recently won the backing of the Committee for Medicinal Products for Human Use (CHMP) for the early use of its drug, Translarna (ataluren), after European regulators had initially slapped down the attempt. And PTC Therapeutics CEO Stuart Peltz heralded Woodcock's remarks by outlining plans to follow up with the FDA.
"In Europe, we had an extensive dialogue with the CHMP regarding the totality of our clinical data and recently received a positive opinion recommending the approval of Translarna for nonsense mutation Duchenne muscular dystrophy," Peltz said in a statement sent to FierceBiotech. "Upon ratification by the European Commission, we expect Translarna will be the first-ever drug approved in Europe as a treatment of the underlying cause of the disease. For the U.S., we have plans to meet with the FDA near-term to obtain additional feedback and discuss potential pathways to bring Translarna to U.S. patients as rapidly as possible."
Time will tell whether the FDA's Woodcock was posturing, but the signs of a fundamental change in approach are hard to ignore. Shares of Sarepta, a volatile stock on any given day, shot up 13% yesterday, but all three of the DMD leaders saw their stock prices slip about 5% today.
- here's the letter from Woodcock