Updated with new information, including comments from Sarepta CEO Chris Garabedian.
CAMBRIDGE, Mass. ( TheStreet) -- Important and welcome news out this morning from Sarepta Therapeutics (SRPT - Get Report). Following a meeting with FDA officials, the company intends to submit an application seeking the approval of eteplirsen for the treatment of Duchenne muscular dystrophy.
Sarepta will submit the approval filing in the first half of 2014, the company said Wednesday.
And with that announcement, Sarepta shares fell -- hard. The stock is down 15 percent to $39.60 but sank as much as 17 percent earlier in the day.WTF? Sarepta's announcement Wednesday raised concerns that FDA, while willing to review eteplirsen, remains unenthusiastic about the limited clinical data available to date. Essentially, skeptics believe Sarepta nagged the FDA into allowing an eteplirsen approval filing to proceed, in part to ameliorate the demands of parents of DMD kids who've been lobbying hard for a speedy drug approval. Here's how Sarepta described the decision to file in its press release this morning: The decision to submit an NDA for eteplirsen in 2014 is based on productive interactions with the FDA in a meeting that occurred this week. That meeting was a follow-up to the FDA's review of two recently submitted summary documents that included data on dystrophin and clinical outcomes from the existing eteplirsen studies. The FDA stated in pre-meeting comments that the Agency is "open to considering an NDA based on these data for filing." The Agency, however, requested additional information related to the methodology and verification of dystrophin quantification. Sarepta believes the requests from the Agency can be addressed and incorporated into an NDA submission in the first half of 2014. From this cautious wording, it does seem as if FDA still has some reservations about the eteplirsen data. That's all Sarepta bears (and weak-handed bulls) needed to send the stock lower. A bunch of analyst downgrades contributed to the sell off. Sarepta CEO Chris Garabedian, in a phone interview this morning, says the company chose deliberately to use cautious language to describe the meeting with FDA and the decision to file eteplirsen. "The FDA has to be very careful with their language and their words, and because they're measured with their words, it requires us to be equally careful," he said. Still, Sarepta chose to announce its intention to seek eteplirsen's approval today, less than a day after its sit-down with FDA, because the agency made it clear that it was ready to review the drug. "The last time we met with FDA to discuss an early filing, we waited for the
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