GSK trial of exon 51 skipping on Ambulatory Boys
Does anyone know what is going on with GSK and the trial on exon 51 skipping? They said the trial will not be conducted this year. Does that mean next year, or not at all? Have they pulled the plug on this trial? If they have, why the heck are they moving forward with the non-ambulatory trial which the FDA has green lighted.
Insight????
Replies to This Discussion
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Permalink Reply by Michelle Gonzales on
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I was told by the research nurse for Dr. Wong, that it is supposed to begin in September. I just recently emailed them to get more information, but haven't heard back.
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Permalink Reply by irishgirl on
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It is firm that it will NOT begin this year - GSK confirmed at least that. I just want to know if they are planning on canning it or starting it in 2011. Seems strange that it was not the talk of the CO conference last week. Ugh to all of this crap. I am so tired of this "no information" crap. If you hear anything, please let me know. How are things going for you all otherwise???
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Permalink Reply by Ofelia Marin on
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It is very unfortunate that they do not say anything about the ambulatory trial. I remember when Prosensa assured us that there will be a Phase III ambulatory trial THIS year. We were all concerned that it did not start "early" 2010 at that point...well, now early 2011 doesn't seem like a bad thing and even that is not probable.
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Permalink Reply by jenn on
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hmm, it seems AVI is still talking exon51 in ambulatory boys, in columbus. we had a conversation about our son max a week ago, they say it is still happening this year, also non ambulatory in columbus for our older boy seems to be still happening, i hope, as we have been working with them recently.
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Permalink Reply by Mindy on
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Where did you see the announcement by GSK that the trial will not move forward in 2010?
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Permalink Reply by Laurie Paschal on
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When I spoke with Dr. Wong at the conference, she all but said the trial would not start this year. The problem is not with GSK, it's with the FDA. They've filed what they need to file, it's just a waiting game now.
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FDA turned them down. The FDA wants more data. GSK is responding by moving forward in the EU, and possibly Canada, only, rather than providing the FDA with what they want.
I'm so glad we have a government agency that will defend our right to die of a terminal disease so vehemently.
And yet I can still eat BPA in my food with no complaints from them at all...
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Isn't this just disgusting? I wasn't this upset in a LONG time. I'm looking into moving out of this country. I am now convinced that any DMD medication will be approved here years after EU or Canada which should not be allowed! Why the h@#%^% doesn't my son have the same right to live as any other boy in other countries?
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That is what is so surprising. The FDA has already approved the trial for the non-ambulatory boys and that trial is already enrolling participants. GSK has said NOTHING other than the ambulatory trial with the same drug will not happen this year. Since the FDA has approved the one trial, it seems to make sense that they are waiting on that data before giving final approval to GSK for the Phase III we are all waiting for. The FDA is VERY wary about approving trial on humans. The FDA has neither approved or flatly denied anything yet - other than this non ambulatory trial. That is a start. We all know Canada and Europe are far more lax in their requirements for new drugs. The US healthcare is primarily run by the greedy pharmaceutical companies and they want to make money. How much do they stand to make on this new drug???
Mindy said:FDA turned them down. The FDA wants more data. GSK is responding by moving forward in the EU, and possibly Canada, only, rather than providing the FDA with what they want.
I'm so glad we have a government agency that will defend our right to die of a terminal disease so vehemently.
And yet I can still eat BPA in my food with no complaints from them at all...
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Permalink Reply by arnold on
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Avii is our best chance right now but folks seem to fixated on GSK
irishgirl said:That is what is so surprising. The FDA has already approved the trial for the non-ambulatory boys and that trial is already enrolling participants. GSK has said NOTHING other than the ambulatory trial with the same drug will not happen this year. Since the FDA has approved the one trial, it seems to make sense that they are waiting on that data before giving final approval to GSK for the Phase III we are all waiting for. The FDA is VERY wary about approving trial on humans. The FDA has neither approved or flatly denied anything yet - other than this non ambulatory trial. That is a start. We all know Canada and Europe are far more lax in their requirements for new drugs. The US healthcare is primarily run by the greedy pharmaceutical companies and they want to make money. How much do they stand to make on this new drug???
Mindy said:FDA turned them down. The FDA wants more data. GSK is responding by moving forward in the EU, and possibly Canada, only, rather than providing the FDA with what they want.
I'm so glad we have a government agency that will defend our right to die of a terminal disease so vehemently.
And yet I can still eat BPA in my food with no complaints from them at all...
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has anyone else heard from the AVI folks recently, like i said earlier, it looks like we will have one son in non ambulatory prosensa, and one in ambulatory AVI trials unless they are still just stringing me along (which is entirely possible )
arnold said:Avii is our best chance right now but folks seem to fixated on GSK
irishgirl said:That is what is so surprising. The FDA has already approved the trial for the non-ambulatory boys and that trial is already enrolling participants. GSK has said NOTHING other than the ambulatory trial with the same drug will not happen this year. Since the FDA has approved the one trial, it seems to make sense that they are waiting on that data before giving final approval to GSK for the Phase III we are all waiting for. The FDA is VERY wary about approving trial on humans. The FDA has neither approved or flatly denied anything yet - other than this non ambulatory trial. That is a start. We all know Canada and Europe are far more lax in their requirements for new drugs. The US healthcare is primarily run by the greedy pharmaceutical companies and they want to make money. How much do they stand to make on this new drug???
Mindy said:FDA turned them down. The FDA wants more data. GSK is responding by moving forward in the EU, and possibly Canada, only, rather than providing the FDA with what they want.
I'm so glad we have a government agency that will defend our right to die of a terminal disease so vehemently.
And yet I can still eat BPA in my food with no complaints from them at all...
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will americans still have access to the drug if it is approved outside the US, like we do deflazacort?
Ofelia Marin said:Isn't this just disgusting? I wasn't this upset in a LONG time. I'm looking into moving out of this country. I am now convinced that any DMD medication will be approved here years after EU or Canada which should not be allowed! Why the h@#%^% doesn't my son have the same right to live as any other boy in other countries?
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