|Sarepta Interim CEO Edward Kaye|
Sarepta Therapeutics ($SRPT), ending a protracted process that may have cost its last CEO his job, submitted its treatment for Duchenne muscular dystrophy (DMD) to the FDA, setting the stage for a possible approval early next year.
The Cambridge, MA, biotech completed the rolling submission process for eteplirsen, its lead asset, and in the process requested a priority review from regulators. The FDA generally accepts applications within 60 days, after which it begins a 10-month review process. If the agency grants Sarepta's request for priority status, that time frame shrinks to 6 months.
The submission puts Sarepta about two months behind BioMarin ($BMRN), which filed its similar DMD treatment in April. The FDA accepted that application Monday, putting the drug, drisapersen, on the priority path and promising to make a final decision by Dec. 27. The rare disease-focused BioMarin bought into the DMD race last year after agreeing to pay as much as $840 million for Prosensa and the once-failed drisapersen.
The next hurdle for each drug is an as-yet-unscheduled date with a panel of FDA advisers, a group that makes nonbinding recommendations on whether the agency should approve submitted treatments.
Sarepta's candidate, designed to treat the rare, muscle-wasting DMD, has had a rocky trip to this point. Eteplirsen successfully delayed the disease's effects in a small Phase II study, but whether those results are enough to merit an early approval has long been the subject of debate. The issue put former Sarepta CEO Chris Garabedian, a bullish defender of eteplirsen, at odds with regulators, whose request for more data delayed the company's planned 2014 submission and deflated the biotech's value.
After ugly boardroom in-fighting came to light last summer, Sarepta dismissed Garabedian in March, saying the change would improve "regulatory discussions and relationships." The board promoted Chief Medical Officer Edward Kaye to take his place with a simple directive: get eteplirsen back on track.
Now, with the drug's data in FDA hands, Kaye has completed the first step of that process.
Sarepta's treatment targets the roughly 13% of DMD patients whose disease stems from a flaw in the gene responsible for producing the protein dystrophin. Eteplirsen works by getting the body to skip over that genetic defect, restoring the flow of dystrophin and alleviating symptoms of DMD.
Sarepta's share value fell about 9% on the news Monday, while BioMarin stayed about flat.
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