'Together, FDASIA and PDUFA V lay down alternative, expedited pathways for drug companies to gain approval for this class of treatments without all the belt-and-suspenders work of multiple, large trials designed to prove efficacy and safety beyond a shadow’s doubt.
One of the prime beneficiaries of this new approach by the FDA could be Sarepta Therapeutics (Nasdaq: SRPT). Sarepta is developing eteplirsen, a novel treatment aimed at a subset of young boys suffering from Duchenne muscular dystrophy (DMD). DMD is the result of a genetic mutation that inhibits a person’s ability to manufacture dystrophin, a necessary component in healthy muscle tissue. Boys who suffer from DMD begin to show symptoms at an early age, and it is a progressive degenerative disease which eventually affects all voluntary muscles in the body. The average life expectancy of a boy born with DMD is 25; there currently is no treatment for the disorder.
In October 2012 Sarepta reported stunning results for a 12-patient phase II study of eteplirsen. The results were so earthshattering that the stock almost tripled the day they were announced. Since that time, the stock has been volatile, dropping from the low 40s to the 20s as Sarepta took advantage of its’ high-flying stock price to raise $125 million from institutional investors and prepare for large-scale commercial manufacturing of eteplirsen.'