Sarepta Drug Helps Boys With Duchenne to Walk; Shares Boom

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the 30 percent of normal threshold that researchers think is necessary to show clinical improvement. The 6-minute walk test, on the other hand, is easy to administer consistently and is a standard goal for FDA-approved clinical trials. That test showed that boys on the Sarepta drug remained pretty stable in their walking ability through 36 weeks, and then started to show stronger walking ability after 48 weeks. Boys in the placebo group were allowed to “cross over” and start taking the experimental drug after 24 weeks, when many of them started to decline in their walking ability. When researchers looked at the muscle biopsies from those boys after 48 weeks, they saw that this group was also able to produce significant amounts of dystrophin (about 38 percent of normal), and that was able to help stop the decline in their walking distances.

Sarepta plans to discuss this latest batch of clinical trial results with the FDA, probably in early 2013, Garabedian says. The company said back in April that it believed its results were good enough to advance the drug into the third and final phase of clinical trials. But since then, Garabedian has suggested he thinks it might be feasible for the company to seek FDA approval based on this small 12-patient study, under the agency’s “accelerated approval” pathway for groundbreaking new medicines.

Regardless of what regulatory pathway is used, Garabedian says the key question will be “what it will take to get this drug approved the most rapidly.”

One key variable will be how much pressure patients put on the FDA to get access to the new medicine, based on this preliminary glimpse at the data. Some parents of boys in this study were so emotionally moved by the improvement they saw, they told local media outlets tales about how their boy could suddenly climb the stairs on the bus by himself after getting the Sarepta drug. In another case, parents talked about how a boy could twist open a milk carton by himself.

Sarepta didn’t encourage the parents to seek media attention on behalf of the drug, and wants to make sure the anecdotes don’t overshadow an evidence-based scientific reading of the data, Garabedian says. But at the same time, those parents and their supporters at patient advocacy groups could end up being powerful allies for Sarepta in its quest to start selling its first marketed product.

“We were already getting demand and requests from patients asking us how quickly we can help them get their hands on this drug,” Garabedian says. “I can’t imagine what the demand for this drug will be from the advocacy community after they see this data.”

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