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?
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!=)
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".