What happened
Shares of Sarepta Therapeutics (SRPT 2.75%) were skyrocketing 24.9% higher as of 10:37 a.m. ET on Monday. The huge gain came after the company announced Friday evening that a U.S. Food and Drug Administration (FDA) advisory committee voted in support of accelerated approval of SRP-9001 in treating Duchenne muscular dystrophy (DMD).
Sarepta said that the FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted 8-6 in favor of approving SRP-9001. DMD is caused by genetic mutations that prevent the production of the dystrophin protein. SRP-9001 is a gene therapy that's designed to correct this underlying genetic issue and help produce dystrophin in the muscle tissue.
So what
An 8-6 vote might seem really close. However, the bottom line is that Sarepta got the positive advisory committee recommendation that it needed.
Investors are fully aware that this vote bodes well for Sarepta's chances to add a fourth approved DMD therapy to its lineup. To be sure, the FDA doesn't have to go along with the advisory committee. However, the agency pays close attention to the opinions of the scientific experts it convenes to review regulatory applications. And it usually does approve therapies that receive a thumbs-up from those advisory committees.
Now what
The FDA has set a PDUFA date of May 29 to make its final decision on accelerated approval of SRP-9001. Sarepta CEO Doug Ingram said that the company's top priority is to work with the agency to complete the review of its regulatory filing.
