Sarepta has requested an end of Phase II meeting with the FDA, which is likely to occur before the end of this month. The focus of this meeting will be the company's request for accelerated regulatory approval on its experimental drug Eteplirsen, which is designed to treat
Duchenne Muscular Dystrophy ((DMD)).
There are many Sarepta bears who believe the drug will not receive early approval, and in my opinion, they are not considering the ever changing political landscape surrounding the FDA. Lawmakers have been putting additional pressure on the FDA to get drugs to market which are designed to treat rare diseases. DMD is a very rare disease that affects children - providing additional pressure for the FDA to grant accelerated approval here.
I wonder if the bears have read Section 902 of The FDA Safety and Innovation Act - "Breakthrough Therapies."
According to author Michael Mccaughan;
The provision creates a new section of the FD&C Act, and inserts it directly before the "Fast Track" and Accelerated Approval sections also included in FDASIA. That symbolic position indicates the intent: something that happens even earlier in drug development than the existing FDA mechanisms.
Also, it will be hard for the FDA to ignore Jenn McNary, mother of a child who suffers from DMD, who has been one of many strong advocates petitioning the organization to allow accelerated approval of Eteplirsen.