PTC Therapeutics, Inc. (NASDAQ:PTCT) investors are having a rough morning after the drug maker announced that the FDA has denies the company’s appeal of the Refuse to File letter it received in February regarding its New Drug Application (NDA) seeking approval of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy. The company plans to escalate its appeal.

PTC Therapeutics shares reacted to the news, tumbling nearly 30% in early trading Monday.

“We believe that fair consideration of the totality of Translarna’s data requires a full review of our application by the FDA,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “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.”

In addition, the company maintains its position that PTC should, under existing law and in fairness to patients, be provided the same opportunity for full review that the DNP gave to other recent applicants for products in development for different subsets of the DMD population.

“I am disappointed that a treatment for patients with nonsense mutation DMD is still not receiving a fair opportunity in front of the FDA,” said Pat Furlong, President and Founder of Parent Project Muscular Dystrophy. “This inconsistency is unacceptable and is concerning for the entire community. 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.” (Original Source)

On the ratings front, PTCT stock has been the subject of a number of recent research reports. In a report issued on October 10, Credit Suisse analyst Alethia Young reiterated a Hold rating on PTCT, with a price target of $15, which implies an upside of 47% from current levels. Separately, on October 3, Merrill Lynch’s Tazeen Ahmad reiterated a Hold rating on the stock and has a price target of $15.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Alethia Young and Tazeen Ahmad have a total average return of -4.4% and 48.4% respectively. Young has a success rate of 40.5% and is ranked #3790 out of 4180 analysts, while Ahmad has a success rate of 57% and is ranked #123.

As of this writing, all the 7 analysts who cover the stock suggest a Hold rating . The 12-month average price target assigned to the stock is $14.00, which implies an upside of 37% from current levels.

PTC Therapeutics, Inc. engages in the discovery, development, and commercialization of orally administered, small molecule therapeutics targeting an area of RNA biology. Its preclinical and discovery programs are focused on the development of new treatments for multiple therapeutic areas, including neuromuscular disease, oncology and infectious disease. It product, candidate ataluren, is used for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation.