Posted by Michael Wonder on 06 Sep 2017
We may soon learn just how flexible Scott Gottlieb's FDA is
6 September 2017 - One the FDA's biggest controversies is coming back to test its new leadership.
Sarepta Therapeutics on Wednesday released promising early data from a study of a new drug to treat some patients with the rare muscle-wasting disease Duchenne Muscular Dystrophy (DMD). The drug is a follow-up to Exondys 51, another DMD treatment the FDA approved last year -- over the objection of some of its own scientists -- to treat a different subset of patients. The company plans to ask the FDA if it can seek accelerated approval of the medicine, according to Leerink analyst Joseph Schwartz. Its shares rose 11% on Wednesday.
No company has benefited more than Sarepta from an FDA shift in recent years toward greater flexibility in approving drugs for rare and deadly conditions such as DMD. And the company may reap more rewards from a new FDA regime that appears open to continuing that shift.
