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Sarepta Therapeutics, Inc. Message Board

  • vskomarovsky vskomarovsky Dec 9, 2013 11:44 AM Flag

    if OK, from stat05

    I do not think that my views are different. My point about PTC and Ataluren is that PTC took a different route and in my opinion did not fight as hard to get their drug approved. Instead, after stopping the phase 2b trial, they took YEARS to start a phase 3. During these YEARS they did not collect additional data on the boys (several) that continue to take Ataluren. That data would have been a very valuable asset to prove to the FDA that their drug continues to keep these boys stable. Moreover, they never reported dystrophin. Why? They took 2 biopsies in their phase 2b. Reporting positive biopsy data would have been another very important asset in their continued FDA discussions. Every piece of positive data and information needs to be used when a company wants to get a drug approved.

    I just think that it takes an effective drug AND a sponsor fighting hard to get a drug approved, to give each drug the highest likelihood of success. In my view, to date, Garabedian is the first pharmaceutical official dealing in the Duchenne field to look at this generation of boys and not see a collection of lab rats available for his experimentation. He is the only one who decided to fight for my son's generation. When their phase 2 trial started they did not have funding to include more boys (the original plan was to have 24 subjects) and no org wanted to provide additional funding. So here we are with a drug that produced dystrophin in ALL boys in phase 2 AND stabilizes them for 2+ years. Not only that, it reversed toe walking in one boy who will turn age 13 in January (this has NEVER been seen before in Duchenne) and improved the 6mw in others (all selected for this trial to have a high likelihood of decline). This is compelling evidence that was not reported in any other Duchenne trial to date and it needs to be reported.

    Just my opinion from what I lived for the past 6 years. I wish nothing but the best for PTC and Ataluren or ANY drug that might change the final outcome for Duchenne boys!

    Sentiment: Strong Buy

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