STAT Plus: As Sarepta awaits approval of new Duchenne drug, big questions hang over therapy already on market
As Sarepta Therapeutics waits for the Food and Drug Administration to announce an approval decision on a new drug to treat Duchenne muscular dystrophy, the biotech is years behind schedule on a requirement to confirm the muscle-function benefit of its first and top-selling medicine Exondys 51.
The FDA has until Monday to decide if Sarepta can market a second drug called Vyondys 53 to treat patients, mostly young boys, with the inherited and fatal muscle-wasting disease. Approval would accelerate Sarepta’s revenue growth because it targets a different genetic form of Duchenne than Exondys 51.
