Sarepta Therapeutics was informed earlier today that an adverse event report was submitted to the FDA’s adverse event reporting system (FAERs), a post-marketing surveillance database for approved therapies. Sarepta states that based on their investigation the report was erroneously submitted to FAERs.
We appreciate Sarepta’s quick response to this report and we will monitor this situation closely and report any additional information to the community. As always, PPMD continues to convene all industry partners, as we know that we still have much to learn in gene and cell therapies.
We are so grateful to the individuals and families who are helping to pave a pathway forward for our community — and we hold their safety as our utmost priority.
Read the Announcement from Sarepta
Sarepta Therapeutics Comments on Erroneous Submission to US FDA Adverse Event Reporting System (FAERS)
August 8, 2019 at 3:27 PM EDT
CAMBRIDGE, Mass., Aug. 08, 2019 (GLOBE NEWSWIRE) — 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.