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????

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Would there be any possibility of the US waiting to hear results from EU or Canada then maybe the US could go to a phase lll trial here?
I guess it's possible. We would need to know the IND process, in other words can you re-file for IND w/o providing the requested data? At this point FDA requested additional data and GSK choses to ignore the request, can they re-file 6-12 months from now providing data from phase III EU trial instead? Then we need to know if they are interested in doing this or they just gave up on the US market until they approve this drug in EU and Canada ~3 years from now (if all goes well).

Tonya said:
Would there be any possibility of the US waiting to hear results from EU or Canada then maybe the US could go to a phase lll trial here?
Seriously, I honestly wanted to know any information about the FDA decision and GSK/Prosensa trials. I just felt like there was information that I wasn't getting and wanted to see if anyone had the facts about what is going on. Okay, I get the picture, it seems like I can certainly get told. I mean of course the FDA has approved "crap", just wanted to ask the community for help with information. Maybe there was a location of information or something that some of us didn't know about. Obviously, I'm not getting the information that many of you are. Anyway, now I will do the usual a blend into the background.
I don't think we're going to get more information. This is the end of the road, information-wise, until Prosensa/GSK chooses to publish. And we're not going to be given an informed choice on whether to participate. The FDA has said no, GSK has done a cost/benefit analysis and chosen to move forward elsewhere. They're in no hurry - they have deep pockets. And their allegiance is to their shareholders, not to the DMD community...

I think that's the frustration that all of us have - we can't get any more information beyond what we've already been told.
FDA never said no trial in the US. May be the want additional data from the non ambulatory trial before allowing the big phase III to begin in the US. As for the IND it is the same drug for the ambulatory and non ambulatory so i think if the IND is open for the non ambulatory it is open for the ambulatory as well. Any thoughts????
I do not think so. Keep in mind that the ambulatory is phase III which means the boys are dosed weekly for a long period of time (1-2 years). The non-ambulatory is a single injection.

FDA requested data and GSK decided it is not a good investment to spend time and money to collect this data. They will conduct the trial outside US. This is just a trial testing if the drug works. There is a chance it doesn't, so GSK interest is to test it and see if it works or not. For them, at this point, it is not important WHERE to conduct the trials. Of course, in the future, if this looks like an effective therapy, they will want to gain access to the US market but that's somewhere in the future when they are certain that this drug has a good chance to be approved. This is how I see it. It's a business.

Ana Vaish said:
FDA never said no trial in the US. May be the want additional data from the non ambulatory trial before allowing the big phase III to begin in the US. As for the IND it is the same drug for the ambulatory and non ambulatory so i think if the IND is open for the non ambulatory it is open for the ambulatory as well. Any thoughts????
I dont quite see why everyone is jumping all over the FDA. If GSK was asked to provide more info and they CHOSE not to, sshouldnt we have the issue with them and wonder just why they chose not to?! Also, as someone said, its a business. The USA is "lawsuit happy", so it makes sense that the fda would be more stringent with approvals, to avoid potential lawsuits if the kids in trials have major side effects. You can sign all the legal papers you want taking the risk and saying you wont sue, but a lwyer can and will find a way around those. Maybe Canada and EU have less stringent rules and regulations and less payouts form lawsuits. Just throwing things off the top of my head. Noone jumo on me please!=)
Hey Samantha, I think those pharma comps lawyers make sure that what parents sign is very clear so there cannot be winable lawsuits. That's all they do, protect the company.

Samantha Dearing said:
I dont quite see why everyone is jumping all over the FDA. If GSK was asked to provide more info and they CHOSE not to, sshouldnt we have the issue with them and wonder just why they chose not to?! Also, as someone said, its a business. The USA is "lawsuit happy", so it makes sense that the fda would be more stringent with approvals, to avoid potential lawsuits if the kids in trials have major side effects. You can sign all the legal papers you want taking the risk and saying you wont sue, but a lwyer can and will find a way around those. Maybe Canada and EU have less stringent rules and regulations and less payouts form lawsuits. Just throwing things off the top of my head. Noone jumo on me please!=)
I think what we're questioning here is whether the additional data is truly necessary in this particular situation and at this point in the drug's development.

I'm not jumping on anyone - really. I just don't trust the FDA's motives or decisions.

Ana - it's a totally separate IND. Each protocol has it's own IND and must be approved separately
Maybe we are all jumping to conclusions which so many of us do here on this site. We cannot help it due to the nature of our situations. We all want the same thing. GSK has made NO formal announcement other than at the conference where they stated this trial would not be this year. GSK needs to come forward and explain themselves and their actions or lack there of. Until that time, all we know has come through the filter of others and not formally announced by GSK.

Mindy said:
I think what we're questioning here is whether the additional data is truly necessary in this particular situation and at this point in the drug's development.

I'm not jumping on anyone - really. I just don't trust the FDA's motives or decisions.

Ana - it's a totally separate IND. Each protocol has it's own IND and must be approved separately
Has anyone heard anything like or similar to this FDA denial idea...

FDA denial might have to do with dosing and toxicity; the 2'Omethyl chemistry is known to be considerably more toxic than the morpholino (actually, the best data on morpholino toxicity was done only very recently, thanks to the DoD, FED, and CureDuchenne that footed the $3 million bill to do it). FDA is usually pretty sticky about having standard animal tox data in hand, supporting the human doses planned, or they freak (e.g. won't approve). I think I mentioned before that I ran an exon skipping workshop in DC about 3 yrs or so ago, and the key FDA rep attended and said straight out: "We don't approve the 2'Omethyl drugs; too much toxicity".
The guy from GSK at the conference did mention some issues with kidneys at higher doses.

irishgirl said:
Has anyone heard anything like or similar to this FDA denial idea...

FDA denial might have to do with dosing and toxicity; the 2'Omethyl chemistry is known to be considerably more toxic than the morpholino (actually, the best data on morpholino toxicity was done only very recently, thanks to the DoD, FED, and CureDuchenne that footed the $3 million bill to do it). FDA is usually pretty sticky about having standard animal tox data in hand, supporting the human doses planned, or they freak (e.g. won't approve). I think I mentioned before that I ran an exon skipping workshop in DC about 3 yrs or so ago, and the key FDA rep attended and said straight out: "We don't approve the 2'Omethyl drugs; too much toxicity".

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