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Hi Mark, I would lke to know if Genzyme is trying to approve the use of Ataluren in Europe.
I don't exactly understand what happens with Ataluren. It's been so long since they stopped the trial and I do not see any clear path even after so may months. Are they collecting more data now through individual INDs? Do they plan to file for market approval? What is the next step? I am quite confused about this. What happens these months, are they still analyzing data? Haven't they said during the first CC after they stopped the trial that the biopsy data will be available by now? This is really incredible, 150 biopsies take so long, even if they would look at one biopsy per day, they should still be done by now. My friend is a pathologist and does a few biopsies per day as part of her job. What's going on here?
I just hope something like this does not happen with the other drugs tested in the future.
I think that maybe the problem is that the results of the biopsies are not good enough. To approve the first drug of the market is easier than to approve the second one. Now the only thing that they have to show is that is better to take ataluren then nothing. But if they see something strange they have to explain it. We know that something strange happens because its not normal that the low dose is better then the high dose. Maybe the results of the biopsies are also "strange" and the FDA will only approve the drug if they find an explanation, and it can take time. But if the drug will be approved for cystic fibrosis I am sure that Duchenne patients will use it too, because PTC demonstrated that the low dose is better than nothing.
I think that maybe the problem is that the results of the biopsies are not good enough. To approve the first drug of the market is easier than to approve the second one. Now the only thing that they have to show is that is better to take ataluren then nothing. But if they see something strange they have to explain it. We know that something strange happens because its not normal that the low dose is better then the high dose. Maybe the results of the biopsies are also "strange" and the FDA will only approve the drug if they find an explanation, and it can take time. But if the drug will be approved for cystic fibrosis I am sure that Duchenne patients will use it too, because PTC demonstrated that the low dose is better than nothing.
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