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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".
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".
Did they not also mention that it was no big deal?????
Laurie Paschal said: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".
I'll believe 2011 when I see it.
If the next round in the EU hasn't started yet, is scheduled to take a year, and needs to be complete and analyzed before the US trials begin, we're looking at 2012 at the earliest....
This is the email we just received from our genetic counselor...
I am writing to update you with news on the exon skipping trial. Here is the update from our research coordinator Amy:
I just got off the conference call with GSK. Basically, the FDA did not approve the study, so they won’t be going forward with it. They are analyzing the data from the small study they did in Ohio and may have something at the beginning of 2011.
It sounded like they are able to do the study in Europe and will be speaking with Canada to see if performing it there is a possibility.
I wish I had better news. I will keep you posted if things change. Let me know if you have any questions. Here is the contact number for GSK to see about European or Canadian trials:
Contact: US GSK Clinical Trials Call Center 877-379-3718
I think he is meaning that the results of the European trial will be included in the package submitted to the FDA for approval of the drug for the clinic, it is not required for the long term trial planned for the US. My understanding is that they are waiting on the completion and analysis of long term animal safety studies to support a long term human trial. (Presumably it is a 6-9 month study, possibly with a 3 month recovery period?). Thus the "live" portion is probably complete and they are in the process of analyzing the data. These types of studies generate a LOT of data and it takes time for the data to be analyzed and conclusions reached, even when there are no adverse effects. If they plan to submit the data later this year (as he states) to support the application for a human trial, it is not unreasonable to expect a long term human trial to initiate in 2011. Having said all that, I understand the frustrations, clinical trials can get delayed for numerous reasons (I have run animal safety studies supporting human trials in the past and have experienced delays first hand), my son would benefit directly from this compound and I am acutely aware of the timing issues and how they impact my son.
Mindy said:I'll believe 2011 when I see it.
If the next round in the EU hasn't started yet, is scheduled to take a year, and needs to be complete and analyzed before the US trials begin, we're looking at 2012 at the earliest....
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