Friday, July 31, 2015 6:48:44 PM
SRPT:
I would agree with your assessment that the drug has a modest effect (I've been of that belief long before becoming a shareholder if one goes back to see my posts) and unfortunately (especially for the kids) they will likely still become non-ambulatory at some time. I will also concede that better care and parent involvement may also be increasing the perceived benefit.
That being said you don't seem open to the idea that FDA would approve a drug with modest efficacy and a benign safety profile for an indication where there are no approved therapies not to mention its a life threatening indication for children. Basically, I don't think the bar needs to be very high. I am also very pleased at what I've publicly seen with the CEO change and the non-confrontational approach with regulators, competitors and seeing this as part of a treatment regimen. These are all positives that Dr. Kaye has done.
Not to bring up the old ERT argument again
(and I am certainly not comparing the efficacy to ERT's) but one important finding that I think may be quite pertinent is that starting treatment very early in the disease and treating longer exaggerates the benefit.
I would agree with your assessment that the drug has a modest effect (I've been of that belief long before becoming a shareholder if one goes back to see my posts) and unfortunately (especially for the kids) they will likely still become non-ambulatory at some time. I will also concede that better care and parent involvement may also be increasing the perceived benefit.
That being said you don't seem open to the idea that FDA would approve a drug with modest efficacy and a benign safety profile for an indication where there are no approved therapies not to mention its a life threatening indication for children. Basically, I don't think the bar needs to be very high. I am also very pleased at what I've publicly seen with the CEO change and the non-confrontational approach with regulators, competitors and seeing this as part of a treatment regimen. These are all positives that Dr. Kaye has done.
Not to bring up the old ERT argument again
(and I am certainly not comparing the efficacy to ERT's) but one important finding that I think may be quite pertinent is that starting treatment very early in the disease and treating longer exaggerates the benefit.
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