Looks solid. I wonder why they couldn't themselves apply to AA. Perhaps, they are playing it safe and comprehensively, due to the adverse side effects (which btw don't seem to offset the drug benefit).
Prosensa has not released any data on their US phase 2 trial, so we don't know if that data would support AA filing. We do know that Prosensa/GSK data from their European trial shows good efficacy but still shows adverse events requiring hospitalization (if those are not SAEs, then I don't know what is). I suspect that the FDA would hesitate to approve AA for their drug when it knows that Sarepta has an effective drug without any drug-related adverse events.
Patriot, The putative "good efficacy" that you report that prosensa released post-PhaseII was without the context of a placebo cohort. Means nothing. Wait for a well-controlled trial before comparing efficacies.
They are considering applying for AA. The side effects so far are not severe. No SAE reported in the first group of 12 now on the drug for 150+ weeks. You could watch the Italy presentation and easily see this. That is the reason why one would put their child on this drug. Remember the alternative of not having a child on one of this drugs?
Solid?? Yes it does, LIKE SRPT's efficacy data. They both make dystrophin, but at what cost you ask? Great question, so................ stick with me cuz this one is the BIG one their (GSK) toxicity issues aren't going away because those idiots chose WAY inferior chemistry, and that is not going to change. Lets see, if I were a parent of a DMD boy, would I want little Jimmy to have blood dyscrasias/thrombocytopenia AND lose his kidneys while making needed dystrophin? Or, would I for some strange reason, want my boy to have healthy blood and retain the use of his kidneys? I'm gonna go out on a limb here, and go with "KEEP THE KIDNEYS". No matter how you paint it, GSK/Prosena's drug is a pig, a losing PIG, and no parent in their right mind will chose said pig over a drug that replaces dystrophin and causes no harm. It's getting harder and harder to spin the GSK/Prosena story in a positive light isn't it "Oneway".. Good luck with that, cuz you're going to need it. And thanks in advance for the massive launch to the share price that you're buddies/bosses with the huge short position are going to cause as they are forced to cover in the very near future.
lol..is that why the parent's of thode in the trial are coming out in droves to tout gsk or are they waiting for the kids to get over all the severe infections they're encountering while on the drug first?