PTC Therapeutics, Inc. (NASDAQ:PTCT) was crushed underfoot investors stampeding for the exits after the company admitted in a regulatory update that the FDA had denied the company’s first appeal of the refuse to file letter issued by the FDA’s Division of Neurological Products (DNP) on February 22, 2016.
The action related to PTC’s New Drug Application (NDA) for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
Shares plunged 39.94% to $7.97, with 12.01 million shares traded, against the average volume of 1.33 million shares.
PTC Therapeutics, Inc. (NASDAQ:PTCT) hints at unfair treatment at the hands of the FDA re: Translarna
“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.”
The company also warned that it intended to escalate the denial of the appeal to the next supervisory level within the FDA and that it may have to go through many such motions at higher and higher levels.
PTC Therapeutics, Inc. (NASDAQ:PTCT) gives another jolt to investors
In a one-two punch, PTC also broke the news that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) had, as a part of its ongoing annual review of the marketing authorization for Translarna, issued a request for supplemental information, including a request categorized as a major objection.
The multiple issues regarding Translarna, from both sides of the pond, understandably triggered investor panic, and caused the resulting downslide.
Unfortunately, the fall has erased the entire recent advance the PTC shares made between August 8 ($7.60) and October 11 ($14.57).