Sarepta to File New Muscular Dystrophy Drug for FDA Approval

Sarepta Therapeutics, a biopharmaceutical company that is the leader in RNA-based medicine, is set to file their new muscular dystrophy drug for FDA approval by the end of this year.

Their new experimental drug, eteplirsen, hopes to provide treatment for those with Duchenne muscular dystrophy, a fatal degenerative disease that affects one in every 3,500 children worldwide. After meeting with the FDA, Sarepta reported that the two sides reached an agreement that would help accelerate the drug's filing process.

Prior to the meeting, Sarepta was asked to conduct a larger late-stage study for the drug, which was expected to delay the approval process by two years. As a result, many became disappointed and Sarepta's shares went down when that news broke late last year. But Monday meeting spurred a 50% increase in their shares early this morning.

"The FDA haven't reversed their decision from November," said Sarepta Chief Executive Chris Garabedian in a telephone interview via this CNBC news article. "They've reinforced the questions and concerns they had of being able to accept an NDA and consider accelerated approval of just the data set they had in-house, but evolved to say, 'If you give us a little more data, it should be fileable.'"

Last November, the FDA told Sarepta that the company's main study (consisting of only 12 patients) for eteplirsen was insufficient to file the drug for approval. As a result, their share price dropped 64% in one day. However, the FDA most likely reconsidered its original ruling because Duchenne muscular dystrophy affects a very small population of the world and no treatment options exist for it.

There are different subsets of the disease, which further narrows the study pool. Eteplirsen is expected to treat a subset of Duchenne muscular dystrophy that is suffered by 13% of boys, while the company continues to work on other drugs that treat different subsets. Nonetheless, investors and patients are hoping for a more expedited approval process because those who suffer from the disease typically do not make it past 30 years old.