Depending on who you ask, things either fell apart for PTC Therapeutics, Inc. (NASDAQ:PTCT) stock holders, or they have gone gangbusters this year. If you got in PTCT stock prior to Feb. 22 (where shares were trading hands for around $30), then I’m sorry for the plunge that more than halved the stock. Those who got in after the collapse, however, have enjoyed gains of nearly 150% … until today.
Those gains in PTCT stock have since dropped to some 60% after today’s harrowing 30% plummet on news that the Food and Drug Administration denied PTC Therapeutics’ appeal concerning its Duchenne muscular dystrophy drug.
The FDA had stuck PTCT with a “refuse to file letter,” and that’s what PTC Therapeutics was appealing. The FDA issues such letters when an application has “omissions of clearly necessary information.”
PTCT is hoping the FDA will come around to giving its Translarna therapy for DMD a “full and fair review,” including an advisory committee hearing in which “clinical experts and representatives of the patient community” alike can give their thoughts on Translarna as a therapy. PTC Therapeutics CEO Stuart W. Peltz stated the following:
“We believe that fair consideration of the totality of Translarna’s data requires a full review of our application by the FDA. In light of this, continuing the formal dispute resolution process reflects our ongoing commitment to work with regulators and the Duchenne community to make Translarna available to nmDMD patients in the United States.”
The company plans to appeal with the FDA, in what could take several cycles of appeal.
Pat Furlong, president and founder of Parent Project Muscular Dystrophy, expressed his disappointment over the FDA’s decision, believing DMD treatment options aren’t getting their fair shake:
“This devastating, muscle-wasting disease cuts short the lives of boys and young men and every day that we wait for treatments, is a day in which muscle function is lost and not regained. As a community, we cannot rest until there are treatments for all the boys and young men.”
Remember, in mid-September, Sarepta Therapeutics Inc (NASDAQ:SRPT) nearly doubled after having its eteplirsen drug approved as the first DMD treatment, but that victory didn’t come without trials.
As of this writing, John Kilhefner did not hold a position in any of the aforementioned securities.
