Pfizer said yesterday it would open the first US trial sites for its experimental gene therapy for a muscle-wasting disorder, after the Food and Drug Administration (FDA) lifted its hold on a late-stage study.
The FDA had put Pfizer’s trial request on hold after the death of a patient in another early-stage study of the therapy for Duchenne Muscular Dystrophy (DMD), which was also paused.
Pfizer said the regulatory clearance came after data reviews and tweaks to the trial to include a seven-day hospitalisation period to closely monitor patients receiving the gene therapy.
The late-stage study had been underway in 11 countries before it was halted. So far, Pfizer has received clearance from countries including the UK, Canada and Taiwan to restart the late-stage study, the drugmaker said.
DMD is caused by changes in a gene, which result in the absence of protein called dystrophin involved in keeping muscle cells intact. It mostly affects boys.
Pfizer’s therapy is designed to deliver a shortened version of the human dystrophin gene.
The drugmaker expects nearly all sites for the global late-stage study to open by the end of June.
Edits by EP News Bureau