Seeking Alpha

The latest chatter on Sarepta's (SRPT -3.7%) etiplirsen: Edison Investment Research's Jason...

The latest chatter on Sarepta's (SRPT -3.7%) etiplirsen: Edison Investment Research's Jason Zhang notes that the FDA is now "squarely in the middle of the bull and bear scenarios" for SRPT and says the regulator will make a decision on the company's NDA application for etiplirsen "likely before the end of the year." By contrast, Leerink Swann said last week the company may "need to run a Phase III trial in 2014."
Comments (2)
  • That may be too late for some of these kids with DMD. The mothers and kids met with all the top FDA officials. The FDA saw first hand the difference between a child taking the drug and one not. In the history of the world, all of these kids have died young. They need to start taking teh drug asap. Most importantly THERE WERE ZERO as in NO ADVERSE EFFECTS from the drug trials. There is nothing to lose. What sense would it make to conduct addittional tests for several years and let these kids die. Give them a chance.
    24 Apr 2013, 01:27 PM Reply Like
  • Agree100%. Unethical to withold this treatment for kids and families with absolutely no alternatives. I too was skeptical of Agency's position given relatively small data set. But then I started listening to some of the parents of these children - to think they have no voice here is niaive. I see subpart H pathway with postapproval commitment for confirmatory trial. But I can't see FDA holding off approval for several additional years for a second pivotal trial. This would not be in anyone's best interest, and considering this is a fatal rare genetic disease, FDA has latitude for making the right choice here.
    24 Apr 2013, 09:36 PM Reply Like
DJIA (DIA) S&P 500 (SPY)