Sarepta Therapeutics (SRPT) says adverse event report was erroneously submitted to FDA's FAERs
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, was informed earlier today that an adverse event report was erroneously submitted to the FDA’s adverse event reporting system (FAERs), a post-marketing surveillance database for approved therapies. Our investigation to date indicates that this report was not submitted to the FAERs database by a Sarepta employee or the study’s principal investigator.
The submission reported a case of rhabdomyolysis in a participant in Sarepta’s Study SRP-9001-102, a blinded, placebo-controlled trial investigating the use of Sarepta’s micro-dystrophin gene therapy candidate in patients with Duchenne muscular dystrophy. Two weeks post-infusion, the patient presented with dark colored urine and elevated creatine phosphokinase (CK) levels but was otherwise asymptomatic. He was hospitalized for observation, discharged the following day and test results returned to baseline.
Study 102 is a one-to-one blinded study and thus a subject presenting an adverse event could be either on active therapy or in the placebo arm of the trial.
While Sarepta and its principal investigator remain blinded to the study, the study drug safety monitoring board is unblinded to the event and has reviewed the issue and recommended the study continue uninterrupted. No stopping rule in Study 102 was triggered.
Rhabdomyolysis is a commonly understood risk associated with Duchenne muscular dystrophy.