Sarepta Soars on FDA Submission for Duchenne Drug

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Sarepta Therapeutics Inc. shares soared in late trading after the biotech company announced plans to begin submitting data to U.S. regulators on a drug for the rare disorder Duchenne muscular dystrophy.

Sarepta will begin submitting a rolling new drug application this week for eteplirsen and should complete its submission by the middle of the year, the company said in a May 19 statement.

Shares of the Cambridge, Massachusetts-based biotech jumped 39 percent to $22.75 in late trading after the market closed in New York.

Sarepta’s path to gaining regulatory approval has been a rocky one. A delay in October, when the FDA asked the company for more data, sent Sarepta’s shares tumbling 32 percent. In April, Chief Executive Officer Chris Garabedian resigned. His successor, Edward Kaye, pledged to improve relations with regulators.

“The FDA said this was the best meeting they’ve had with Sarepta,” Kaye said in a telephone interview. “It was validation that we could turn it around. Rather than arguing with them on what they needed, we made sure we gave them exactly what they asked for.”

Sarepta has applied for priority review from the FDA, according to the company.

Duchenne muscular dystrophy, a rare disorder that affects boys, causes muscle degeneration and death, often by their mid-20s. Duchenne occurs in about one in every 3,500 boys who are born, according to Sarepta.

There are no approved drugs on the market that halt or reverse the muscle damage, only therapies to help patients manage their symptoms. Sarepta and BioMarin Pharmaceutical Inc. have been racing to be the first to get a disease-modifying drug to market. BioMarin, based in San Rafael, California, completed its regulatory submission on April 27.

(Source corrects statement to show it awaits FDA approval of priority review in sixth paragraph of story that ran May 19.)
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