Date: Jan 15, 2016 Author: Don Seiffert Source: bizjournals (
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Sarepta Therapeutics, the Cambridge biotech whose drug for Duchenne muscular dystrophy will be reviewed next week by an FDA advisory panel, saw its shares cut in half this morning after the agency released documents that appear very critical of the drug's effectiveness.
Sarepta shares were down more than 50 percent Friday morning to $14.99 as of 11:30 a.m., their lowest point since May 2015.
Most observers were not expecting the documents to be released until early next week, and were expecting them to be more positive toward potential approval of the drug. The FDA's executive summary of the documents begins, "The data, overall, did not provide statistical evidence to support the efficacy of etepliresen (the drug) in patients."
The fact that the 12-patient trial did not have a placebo controlled arm made the data on patient's performance on the six-minute walk test -- considered key evidence of the drugs' effectiveness by the FDA -- was called "statistically uninterpretable."
The full documents can be found on the FDA's website
