Can this rare-disease drugmaker deliver?
One biotech company that’s been potentially overhyped for years is Sarepta Therapeutics, a drug developer aimed at tackling Duchenne muscular dystrophy (DMD), a degenerative muscle disease that attacks children, teens, and young adults.
Sarepta’s back and forth with the Food and Drug Administration was the stuff you find on daytime soap operas. On one hand, its phase 2b extension study involving Exondys 51 (which is now a Food and Drug Administration-approved drug for exon 51-skipping DMD) demonstrated a clear benefit via the six-minute walk test (6MWT) over the placebo. On the other hand, the FDA had a hard time making the connection that an increase in dystrophin production and the positive 6MWT benefits were the result of Exondys 51. Eventually, the FDA conceded and approved Exondys 51 with the condition that Sarepta run a phase 3 confirmation study.
