Rare Daily Staff

Sarepta Therapeutics said that it was informed that an adverse event report regarding its experimental gene therapy for Duchenne muscular dystrophy was erroneously submitted to the U.S. Food and Drug Administration’s adverse event reporting system.  

The company’s investigation found that the report was not submitted to the database by a Sarepta employee or the study’s principal investigator.

The submission reported a case of rhabdomyolysis in a participant in Sarepta’s blinded, placebo-controlled trial investigating the use of its micro-dystrophin gene therapy candidate in patients with Duchenne muscular dystrophy. Two weeks post-infusion, a seven-year old patient presented with dark colored urine and elevated creatine phosphokinase levels but was otherwise asymptomatic. He was hospitalized for observation, discharged the following day, and test results returned to baseline.  The incident occurred in February.

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. Sarepta and its principal investigator remain blinded to the study, but the study drug safety monitoring board is unblinded to the event. It has reviewed the issue and has recommended the study continue uninterrupted.

Rhabdomyolysis is a commonly understood risk associated with Duchenne muscular dystrophy (DMD), a rare genetic muscular disease that robs afflicted children of the use of their muscles, eventually leading to difficulty breathing and swallowing. There is no cure for DMD. Currently, Sarepta’s Exondys 51 (eteplirsen) is one of the only approved treatments for DMD.

Pfizer is also developing a DMD gene therapy, and reported in June that in a small trial, one of the first six people dosed was hospitalized with acute renal injury. 

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