Sarepta Rare Disease Advisory Panel Meeting today, page-8

Another interesting read today in Forbes-

...The most powerful argument for voting yes was the presence of all 12 boys who took part in the clinical trials, 10 of whom were still walking after 4 years after beginning treatment with Eteplirsen. It is clear that all 12 boys, even the 2 who could no longer walk, were doing much better than anyone expected based on the normal progression of the disease.
When you consider the fact that in all the years of clinical trials, Eteplirsen has proven to have an excellent safety profile, it is hard to accept that rejecting this drug is the right decision.

Why would anyone want to deny DMD patients the use of a drug that everyone agrees is safe? Yet, the FDA might now stand in the way of patients who want to use this drug because it has not yet been proven effective.

For this reason, the idea that the FDA should only approve drugs that are safe and effective deserves re-examination.

Perhaps the FDA should grant two kinds of approvals, one for safety and another for
efficacy.

Imagine if the advisory committee had been asked to vote on whether Eteplirsen is safe to administer to DMD patients at the dosages used in the trial.

I think this would have easily passed by a large majority. If the FDA were empowered to approve a drug that was deemed safe but not yet proven effective, then the decision about whether to use the drug would be placed where it should be, with patients and their doctors.

http://www.forbes.com/sites/kenkam/...mpaign=yahootix&partner=yahootix#6ec18d236ada