Slow as frozen molasses. What we need is some kind of zone for experimental medicine. A place where people can go to take experimental treatments, or treatments on the way to validation like etiplirsen, if they are willing to bear the risk. Risk would have to be fully and explicitly disclosed to the most extent possible.
This would be good for patients and would stimulate the American biotech industry which is languishing under the heavy thumb of the FDA. Results from these types of interventions could even be combined with normal trial data to shorten the normal onerous FDA process.
So you want us to go back to the 1930s, 40s era when the snake oil medicine was prevalent that killed thousands of children, men and women while the fraudulent companies were making money....
GSK drug, despite spending big money and conducting ethical trials with large sample sizes, the drug failed, which is in the same class of drugs as etiplirsen
And you want eti to be approved with 12 patient data??? MORON, crawl back to where ever you came from.....
Moron, that's like the kettle calling the pot black. For a change instead of saying 12 patients isn't enough how about explaining the the difference between the natural history of the diease and the 12 patients receiving eteplirsen in a logical no nonsese manner. Can't wait to hear from you!!!!
The glaring problem for Eteplirsen with FDA review is the exploratory Phase 2 was done in A SINGLE CENTER - at Jerry Mendell's lab - and that's it. Could Mendell's data be repeatable in another study center? There is no way to know. In decades of FDA approval history, there is been ZERO cases for a single center trial to serve as the pivotal for the approval process.
This is in addition to the 12 patient sample size, with the removal of two patients, for statistical references. The unbinding of the exploratory phase II took place at 24 week time point, and the 6MWD did not hit statistical significance until 36 week after the removal of the 2 non-ambulatory boys for the baseline reference.
The muscle biopsy data is also highly problematic. First and foremost, the baseline along with 12/24 measurements were done with the bicep muscle, while the 48 week was done with the deltoid muscle. This fact was not disclosed by the company until the formal publication of the study over Annals of Neurology, almost 48 weeks after the much touted success in October 2012. The dystophin data analysis done by the GSK/Prosensa group clearly showed different muscles have different dystrophin baseline and that variation existed even within the same donor. In other words, the 48 week dystrophin data for Eteplirsen is GARBAGE and can not be used for any regulatory decision purposes. Then there is the problem of counting positively stained fibers. This is a real problem with the revertant fibers that are formed after the treatment of the drug. These guys didn't use the IMF intensity assay for a reason, and no way by just counting fibers will get through the FDA reviewers.
Now they are setting up the scene for a OPEN LABEL Phase III with a placebo arm where the patients KNOW for sure the drug would not work for them. How ridiculous! Why waste time for the P3 then? They think the FDA is stupid. Why tell the advocacy groups for this design first? To pressure the FDA?