Markets reacted accordingly on Tuesday as PTC Therapeutics (Nasdaq: PTCT) suffered the latest blow to its hopes to gain approval in the USA for Translarna (ataluren) as a treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
The US drugmaker saw its share price slide by 14% to $16.81 after the publication of an FDA report that raises concerns over the data submitted to show Translarna’s efficacy in nmDMD patients, who make up around 10 to 15% of those with DMD.
"Ultimately, no positive results from any prospectively planned analyses that are persuasive"
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