PTC Therapeutics Inc. (NASDAQ: PTCT) was leading the bears early on Tuesday following the announcement that it received a Refuse to File letter from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for Translarna (ataluren).
The FDA said in the letter that the application was not sufficiently complete to permit a substantive review. PTC first learned of the Refuse to File decision via this letter and is reviewing its content to determine the appropriate next steps.
Translarna is an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in DMD.
DMD is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm and heart muscles. Patients with DMD lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.
Prior to this current move in the stock, PTC Therapeutics shares were down by nearly 50% in just the past 52 weeks. However this number shrinks in 2016 as the stock is only down about 13% year to date.
Shares fell more than 46% to a new 52-week low of $15.07 Tuesday morning. The 52-week high is $78.72, and the consensus analyst price target is $78.90.
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