FDA schedules separate meetings to review BioMarin, Sarepta Duchenne drugs
- The FDA will review BioMarin's drisapersen on November 24 and Sarepta's eteplirsen on January 22, 2016. Both drugs are intended to treat Duchenne muscular dystrophy (DMD).
- The FDA has raised some eyebrows by scheduling panel meetings two months apart for drugs designed to address the same rare disease.
- DMD is a rare genetic disease which mainly affects young men. Because the body does not produce dystrophin in those affected by DMD, muscle tissue does not function properly, the body weakens and eventually the patient is paralyzed and dies. Most patients die before age 30.
There has been a great deal of controversy and activity around gaining approvals for treatment of DMD. Numerous parents and other activists have pushed the FDA to be flexible with respect to study design (there were only 12 patients in the eteplirsen trial) and accelerating the approval process.
A group of parents even recruited experts to draft the DMD drug development guidelines. Now, they are waiting for the first review in November—and anticipating, or at least hoping for, a positive response. Some onlookers speculate that BioMarin may have the advantage because it is being reviewed first.
The thinking is that once the unmet medical need is met, it's not as urgent—which is bad for the second company trying to advance a drug for the same disease. However, it's not clear what the outcomes of the panel will be, so at this point the situation is still quite fluid.