MHRA Halts Dosing on Sarepta DMD Trial Over Safety Concerns
February 9, 2018
Rare Daily Staff
Sarepta said the Medicine and Healthcare products Regulatory Agency halted dosing in its late-stage U.K. study of golodirsen, an experimental therapy for to treat Duchenne muscular dystrophy drug involving exon 45 or 53 skipping.
The study continues to enroll and dose per protocol in all other participating countries.
EP Vantage first reported the halt after a post on Facebook from a parent of a child enrolled in the Essence trial due to concerns that a patient in the study developed rhabdomyolysis, a breakdown of damaged skeletal muscle. It is no known whether the rhabdomyolysis was a response to the drug or part of the course of the patient’s disease.
Sarepta said that the U.K. site temporarily stopped dosing because of a single adverse event “that could possibly be related to the investigational drug product.”
“The study remains blinded and the adverse event observed is consistent with those seen in patients with DMD,” the company said in an emailed statement.
The company noted that an independent Data Monitoring Committee reviewed all safety data from the trial and determined that dosing could continue for all patients. “However, because U.K. specific stopping rules of the study, the Medicine and Healthcare products Regulatory Agency required that dosing stop at all U.K. sites,” the company said.
Sarepta said it is currently submitting an amendment to the MHRA. Following approval from the MHRA, dosing can be reinitiated at the UK sites.
February 9, 2018
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