Shares of Sarepta Therapeutics Inc. fell almost 13% just after noon ET Aug. 8, the result of a report that its muscular dystrophy gene therapy caused an adverse reaction in a boy enrolled in a clinical study.
Cambridge, Mass.-based Sarepta is studying SRP-9001 in male patients ages 4 to 7 years old over 48 weeks, plus a 96-week extension study, according to the U.S. Food and Drug Administration's clinical trials database.
An incident submitted to the FDA's adverse event reporting system for approved therapies said the patient developed rhabdomyolysis, a serious illness associated with Duchenne muscular dystrophy. Rhabdomyolysis is a rare disease that causes rapid death of muscle tissue and can lead to kidney damage or failure.
Symptoms of the disorder showed up in the boy two weeks after infusion. He was hospitalized and discharged a day later.
Sarepta issued a release saying the report was erroneous and that none of its employees or study investigators had submitted the data. The study is double-blind, meaning neither patients nor Sarepta and study leaders know which treatments are being given to each individual. Neither side knows whether the boy had been taking the treatment or the placebo.
A safety monitoring board that is not blinded to the study has recommended the study continue.
Trading for Sarepta was halted around 12:30 p.m. ET and shares were down 6.52% to $132.98 at the end of the day.
Sarepta, which reported its second-quarter earnings a day earlier, had recently expanded the study's enrollees from 24 to 40 patients. CEO Doug Ingram said results should be ready by the end of 2020.