The U.S. Food and Drug Administration (FDA) has placed a phase I trial of a CD19-specific chimeric antigen receptor (CAR) T-cell therapy on clinical hold and is requesting additional chemistry, manufacturing, and control information in support of the trial application.
The product, developed by Ziopharm Oncology, uses a new platform called “Sleeping Beauty” that allows CAR T cells to be manufactured and administered within two days – drastically reducing the shipping and manufacturing time of other CAR T-cell approaches. The halted trial would evaluate CD19-targeting CAR T cells for the treatment of patients with relapsed or refractory CD19-positive leukemias and lymphomas.
The FDA appears to be exercising an abundance of caution in the wake of deaths related to other companies’ CAR-T therapy trials. Ziopharm CEO Laurence Cooper, MD, PhD, indicated that he still expects the trial to move forward. “We know what is needed to address the hold issues,” he said, “and are looking forward to responding to the agency in a timely manner.”