Sarepta, a pharmaceutical company, lost a major battle in its quest to gain approval for eteplirsen, its drug for Duchenne muscular dystrophy (DMD).

A U.S. Food and Drug Administration (FDA) panel of outside experts voted 6 to 7, saying that the company was not able to provide significant evidence that the drug boosts the production of dystrophin to a level that provides a clinical benefit.

The committee also voted 3 to 7 against finding substantial evidence that eteplirsen is effective in treating people with the degenerative disease. Three panelists abstained from voting.

"We would like to thank the hundreds of patients and families who participated in the discussion today, underscoring the critical unmet need of people living with Duchenne," Dr. Edward Kaye, chief medical officer and interim CEO of Sarepta, said.

"We appreciated the opportunity to present our data to the advisory committee panel and will continue to work with FDA as they complete their review of the eteplirsen NDA. Today more than ever, we remain committed to our mission of bringing a treatment to the Duchenne community," he added.

Sarepta Shares Tumbling

It is unclear whether the disapproval of the drug has an impact on the shares of Sarepta. The company's shares, however, have been sliding down the slope as much as 46 percent after the FDA advisory committee voted against Sarepta's DMD drug. The drop sent the shares to its lowest in about four years.

The vote came after a gruelling 11-hour session, during which patients and drug advocates aired an emotional voice asking the regulatory agency to accelerate the approval of the drug's marketing in the U.S.

Sarepta, however, has fallen short on important questions regarding eteplirsen's ability to treat DMD, which the advisory committee considered.

"I was not convinced the data was there to prove something based on one, poorly controlled study," said Professor Richard Kryscio of the University of Kentucky, who voted against the drug.

Scientific Data Fell Short On Proving Efficacy

The patients suffering from the disease and their families attended the FDA meeting. They applauded the study's scientists and rebuked FDA regulators.

With more than 50 speakers who talked to the FDA during a public comment, they were all hopeful that the drug would be approved. Much to their dismay, the panelists acknowledged the anecdotes, but believe that the results claimed by the patients themselves were not reflected in the data presented by Sarepta.

"Unfortunately, what I would consider meaningful evidence from the testimony of the families is not properly measured in the study," said Dr. Chiadi Onyike of Johns Hopkins University.

What Is Duchenne Muscular Dystrophy?

DMD is a degenerative neuromuscular disorder causing severe progressive muscle loss and even premature death. It is one of the nine types of muscular dystrophy and it is mainly caused by the absence of dystrophin, an important protein that helps keep the muscle cells intact.

Symptoms of this disease first appear during childhood, usually between ages 3 and 5. It primarily affects boys, but can also affect girls in rare cases.

Patients affected with DMD usually do not survive beyond their teen years, but thanks to advancement in medicine and cardiac care, the life expectancy of boys affected by the condition is increasing dramatically. Some of the patients have reached young adulthood, with some having families of their own and others living into their 40s and 50s.

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