|Former Sarepta CEO Chris Garabedian|
The FDA has given Sarepta Therapeutics ($SRPT) the go-ahead to start a rolling submission for its Duchenne muscular dystrophy drug (DMD), eteplirsen. The green light comes 7 weeks after Sarepta parted company with its CEO Chris Garabedian--and his interim successor was quick to frame the regulatory breakthrough as the result of the subsequent shift in strategy.
Cambridge, MA-based Sarepta will start filing parts of the submission this week and expects to have everything with FDA by the middle of 2015. The timeline puts Sarepta in contention to have its DMD drug assessed by an advisory panel alongside drisapersen, the rival candidate that BioMarin Pharmaceuticals ($BMRN) acquired in its $680 million takeover of Prosensa. Both drugs have followed tumultuous paths to this point, with major, as yet unanswered, questions about the data emerging as they have progressed through development.
At Sarepta, a frosty relationship with FDA has added to the uncertainty. These issues played a role in the departure of Garabedian. Edward Kaye took over in an interim capacity at the end of March and committed to improving regulatory discussions and relationships, an objective he thinks has been achieved. "The FDA said this was the best meeting they've had with Sarepta. 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," Kaye told Bloomberg.
After nearly three years of dizzying ups and downs, investors were just pleased to see the finish line inch into view. The stock jumped 38% in after hours trading to move above the $20 a share mark last seen in October. A lot still rests on the stance of the mooted FDA advisory panel, which the regulator could convene later this year to offer an opinion on the questions hanging over drisapersen and eteplirsen.