Another Duchenne muscular dystrophy gene therapy trial has been held up by the FDA following a serious adverse event. The agency put the brakes on Solid Biosciences’ clinical trial for its DMD treatment following a serious adverse event in one patient.
The latest child patient to be dosed with the microdystrophin gene therapy, SGT-001, experienced a low platelet count, a decrease in red blood cell count, acute kidney injury and cardiopulmonary decline. The patient is in recovery and the five other patients treated in the study are “all doing well,” the company said.
The agency previously halted the trial twice before due to reported serious adverse events.
Pfizer and Sarepta Therapeutics are also developing DMD gene therapies. Pfizer reported similar serious adverse events in a single patient in June, including acute kidney injury and reduced platelet count. The other five patients in the trial showed immune responses as expected, Pfizer said.