FDA asking for another biopsy b4 $SRPT approval makes alot of sense maintaining dystrophin over 2 years would clearly show drug is working— Dan Rosenblum (@sharkbiotech) September 9, 2013-- On a related note, Garabedian dissed DMD rivals Prosensa (RNA) and GlaxoSmithKline (GSK - Get Report) by pointing out the "low bar" used by the companies to assess dystrophin production in the "DEMAND-2" study of drisapersen. [Three different tests were used, but only one had to be positive.] In August, Glaxo posted data showing dystrophin increases from baseline in 72% of DMD boys treated with 6 mg of continuous drispersen and 59% of DMD boys treatedwith 6 mg of intermittent drisapersen. We'll see a more detailed look at the drisapersen dystrophin data from the DEMAND-2 study in October at the World Muscle Society meeting. -- Sarepta has scheduled a meeting with FDA in October to discuss the requirements for the CMC (chemistry, manufacturing and controls) section of the planned eteplirsen approval filing. A hoped-for CMC meeting in September wasn't possible, Garabedian said. Sarepta's guidance for an eteplirsen FDA filing in the first half of 2014 assumes a "reasonable amount of flexibility" from FDA regarding CMC requirements, said Garabedian. This includes stuff stability testing data and comparability of eteplirsen made at small scale versus larger scale. -- On Sarepta's R&D efforts to develop additional exon-skipping drugs, animal testing for an exon 45 skipping drug is underway and similar studies for an exon 53 skipper will be started later in the third quarter. The company continues to guide towards filing INDs for two new drugs next year. -- Sarepta is still blocked in Europe by Prosensa's drisapersen patent but Garabedian said the appeal process continues. Sarepta plans to meet with European regulators at the end of the year to discuss the feasability of an approval filing, even if it means the risk of being sued for patent infringement. "When you have a good drug for a devastating disease that is good for patients, we ill do everything we can to make that drug available," Garabedian said. -- Reported by Adam Feuerstein in Boston. Follow @AdamFeuerstein
Sarepta CEO: Next Eteplirsen FDA Meeting in October
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