Erroneous Adverse Event Report Doesn’t Stop Study: Sarepta

Sarepta Therapeutics says an adverse report that was erroneously submitted to the FDA adverse event reporting system on a case of rhabdomyolysis in a participant in a study of the company’s micro—dystrophin gene therapy candidate in patients with Duchenne muscular dystrophy did not result in any interruption in the study. The company says the report was not submitted by a Sarepta employee or the study’s principal investigator.

The company statement says the patient was hospitalized for observation, discharged the following day, and test results returned to baseline. While the company and its principal investigator remain blinded to the study, the statement says, the study drug safety monitoring board is unblinded to the event and has recommended the study continue uninterrupted.

Rhabdomyolysis is a commonly understood risk associated with Duchenne muscular dystrophy, Sarepta says.