Shares of Sarepta Therapeutics plunged by as much as 28 percent yesterday after the Cambridge company released data showing that patients taking its drug for Duchenne muscular dystrophy showed a progression of the muscle-wasting disease after nearly three years of use.
CEO Chris Garabedian said he still expects the Food and Drug Administration to approve eteplirsen next year based on the results, which showed a decline in walking ability at a rate slower than would be expected in DMD patients. Kimberly Lee, an analyst with Janney Montgomery Scott, said it would be difficult for eteplirsen to get accelerated approval because only six patients in the trial were on the drug.
The precipitous drop in the company's stock is not surprising, Lee said, because investors were expecting to see a stabilization of the disease.
"Instead, the disease is progressing," she said.
But Jenn McNary of Pembroke, whose son, Max, 12, was included in the trial and whose son, Austin, 15, was not, said she has seen firsthand the difference eteplirsen can make. Max can still walk and feed himself, she said, whereas Austin has lost the ability to lift a glass to his mouth and to move himself from his bed to his wheelchair.
"This drug was not meant to stop the disease's progression," McNary said. "What it was meant to do was to turn this serious form of muscular dystrophy into a milder form."
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