Sarepta plays nice with the FDA, sets out plans for second accelerated Duchenne pitch by year's end
Attempting to mend a rather tense relationship with regulators, Sarepta has wrapped up talks with the FDA on how it should move forward with its new Duchenne muscular dystrophy drug: golodirsen. With notes from the FDA in hand, Sarepta $SRPT says it plans to seek rapid approval, with an NDA filed by the end of this year.
The Cambridge, MA-based company stirred some controversy at the agency back in 2016, when the FDA approved Sarepta’s first DMD drug Exondys 51. Some regulators were skeptical of the drug’s benefit to patients, including Ellis Unger, director of the Office of Drug Evaluations. At the time, Unger warned that Exondys 51 was “essentially a scientifically elegant placebo.” He was overruled, however, and the drug came to market later that year. It’s now a decent revenue driver for the company, bringing in $57 million in Q4 sales last year.
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