What: Shares of PTC Therapeutics (NASDAQ: PTCT ) , a biopharmaceutical company focused on developing oral small-molecule drugs designed to treat serious and life-threatening diseases and disorders, dipped as much as 17% after PTC today provided an update on the opinion of the Committee for Medicinal Products for Human Use, or CHMP, for ataluren as a treatment for nonsense mutation Duchenne muscular dystrophy, or nmDMD. However, shares rebounded ended the trading day nearly 3% in the green.
So what: According to a PTC Therapeutics press release, the European Medicines Agency committee has adopted a negative opinion on the company's marketing authorization application for ataluren as a treatment for nmDMD. As the press release states, this opinion isn't a surprise, as the company is still conducting a confirmatory phase 3 trial that will encompass 220 patients and take 48 weeks. Top-line data from this study is expected by mid-2015. PTC still intends to request a reexamination of the CHMP's opinion next quarter.
Now what: The press release pretty much tells you everything you need to know as an investor: the CHMP has a negative opinion on ataluren at the moment; this wasn't a surprise at all given PTC's previously cautious guidance on a marketing authorization approval in Europe; and that it'll simply move forward with its late-stage trial. Other than the fact that PTC's shares have shot to the moon lately and today's news simply gave shareholders a reason to take a few dollars off the table, this CHMP opinion didn't really change the scope of ataluren's development much, if at all.
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