FDA panel meets to discuss muscular dystrophy drug

25 Apr, 2016 10:35 pm

NEW YORK – Hundreds of patients and advocates packed a hotel ballroom in Hyattsville, Maryland on Monday to try to persuade advisors to the U.S. Food and Drug Administration to support approval of an experimental drug to treat Duchenne muscular dystrophy.

In briefing documents last week the FDA reiterated a negative earlier assessment of the drug, made by Sarepta Therapeutics Inc. It questioned the validity of the clinical trials and said it was unable to draw reliable conclusions about the drug’s efficacy

Dr. Billy Dunn, director of the FDA’s Division of Neurology Products, opened the proceedings on Monday by emphasizing that the FDA has not yet made its decision as to whether to approve the drug, eteplirsen. He also addressed patients, assuring them that their voices have been heard.

“It is not the volume of the message but the content,” he said. “We listened, and we listened closely.”

He also laid out the FDA’s responsibility under the law to ensure the drugs it approves are effective. And he urged the panel to make its decision based on science in what could be an emotional meeting.

“Anecdote and emotion do not change the data,” he said.

Duchenne’s is a rare and devastating genetic disorder characterized by progressive muscular weakness and degeneration. It is caused by a lack of dystrophin, a protein needed to keep muscles healthy and primarily affects young men.


The disease typically emerges in childhood, causing weakness in the arms and legs and eventually the lungs and heart. Patients typically lose the ability to walk during adolescence and frequently die in their 20s or 30s, according to the National Institutes of Health.

Sarepta’s senior vice president of regulatory affairs, Shamim Ruff, acknowledged that the company had not produced a “traditional” data set since the company did not conduct a randomized, controlled clinical trial, the gold standard.

Instead it measured the progress of patients in the trial against how patients with the disease progress historically. But Ruff said the benefit of the drug as measured by the production of dystrophin, and clinical benefit as measured by a six-minute walk test, was strong enough to warrant accelerated approval for the drug.

The panel will vote at the end of the day on a number of questions, including whether results of the study provide substantial evidence that etiplirsen is effective. –Reuters




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