When Dr. Ed Kaye was the CEO of Sarepta Therapeutics, he steered the company through a controversial approval process for its drug to treat Duchenne muscular dystrophy. The drug, Exondys 51, won approval based on a trial in only 12 patients, against the advice of a Food and Drug Administration panel and amid strong lobbying by families affected by Duchenne muscular dystrophy.
That decision in 2016 has helped shape FDA policy for rare diseases ever since. Kaye, who’d been Sarepta’s chief medical officer for six years before stepping into the CEO role for nine months, left the company in 2017.
