- In August, the FDA granted priority review status to Sarepta Therapeutics' eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD).
- The FDA wanted larger trials and more data, so Sarepta used data from patients in other trials to create a control arm—and bolster its chances of approval for eteplirsen.
- The problem with gaining approval for eteplirsen has been related to its small trial size. There were only 12 patients enrolled in the study that Sarepta wanted to use to gain approval.
DMD is a rare genetic disease, which mainly affects young men. Because the body does not produce dystrophin in those affected by DMD, muscle tissue does not function properly, the body weakens and eventually the patient is paralyzed and dies. Most patients die before age 30. The fact that it is a rare disease is one of the factors that makes it difficult to enroll a large number of patients.
However, clinical trial results have been positive, with notable comparative improvements in the 6 minute walk test (6MWT) in eteplirsen-treated patients and data showing changes in key biomarker production in these patients. Overall, treatment with eteplirsen significantly slowed disease progress, while improving key pulmonary and ambulatory endpoints.
Now what's needed is new safety data, which Ed Kaye, Sarepta's interim CEO, is determined to deliver. An FDA committee will be continuing review of eteplirsen on November 24. At this point, investors are positive and the stock is up, currently trading at $38.84.