Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne typeWIKIMEDIA; CDC, DR. EDWIN P. EWING, JR. The US Food and Drug Administration (FDA) today (September 19) announced its first approval of a drug for Duchenne muscular dystrophy (DMD). Sarepta Therapeutics’s eteplirsen is “specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD,” according to the agency.
In April, an FDA advisory committee voted 7-to-3 against approval of eteplirsen. Some committee members indicated that they were not convinced of the drug’s efficacy based on the results of a 12-person clinical trial.
The agency OKed eteplirsen, which will be marketed as “Exondys 51,” through its accelerated approval pathway. While the drug will now be available to applicable DMD patients, Sarepta must still verify its efficacy. “Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval,” Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said in the statement.
“Woodcock overruled others in the FDA ...
