Duchenne drug gets FDA approval in “unprecedented” ruling

pharmafile | September 20, 2016 | News story | Manufacturing and Production, Sales and Marketing Duchenne Muscular Dystrophy, FDA, sarepta 

The FDA has gone against the advice of its own staff to approve the first treatment of Duchenne muscular dystrophy (DMS) in Sarepta’s Exondys 51.

DMS is a rare form of muscular dystrophy affecting only one in 3,600 boys. The “unprecedented” move comes after nine months of delays for the treatment as the FDA attempted to determine its clinical benefits and three other candidates were rejected. The decision was thought to be influenced by the actions of hundreds of patients and their families who lobbied to push for a treatment to be made available. The drug could now be available as early as the end of the year.

The ruling also requires that Sarepta conduct a two-year study to confirm the efficacy of the treatment at improving motor function in Duchenne patients losing the ability to stand and walk.

“This decision is extremely unprecedented and follows major disputes among agency personnel regarding their interpretations of very limited . . . data,” biotech analyst Joseph Schwartz at the Boston health care investment bank Leerink Partners commented. “Ultimately, it appears to us that the FDA bowed to external pressure from patient advocates and others who demanded that a safe and potentially efficacious drug be made available.”

The Oregon-based company’s shares shot up almost 74% to $48.94 following the announcement.

Matt Fellows

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