Novartis‘ CAR T-cell therapy CTL019 has received breakthrough therapy status by the U.S. Food and Drug Administration for the treatment of adult patients with relapsed and refractory diffuse large B-cell lymphoma (DLBCL) who failed two or more prior therapies.
This is the second indication for which CTL019 has received this designation, which is meant to expedite the development and review of the investigational drug. The first was for the treatment of pediatric and young adult patients with relapsed and refractory B-cell acute lymphoblastic leukemia, and the company has filed a marketing application for this indication.
“At Novartis, we are eager to unlock the full potential of CTL019, including the potential to help patients with r/r DLBCL,” Vas Narasimhan, global head of Drug Development and chief medical officer of Novartis, said in a press release.
“We look forward to working closely with the FDA to help bring this potential new treatment option to patients as soon as possible.”
CAR T-cells are genetically engineered to recognize and attack a particular tumor. They are harvested from a patient and taken to a lab, where researchers modify them to target a specific cancer protein.
The University of Pennsylvania developed CTL109 to recognize the CD19 protein, which is widely expressed in lymphoma and leukemia cells.
The FDA designation was based on data from the multi-center JULIET Phase 2 trial (NCT02445248), designed to evaluate the safety and effectiveness of CTL019 in adult patients with relapsed and refractory DLBCL. The study enrolled 130 patients.
The study’s primary objective was the proportion of patients who responded to the therapy, and secondary outcomes include safety, time to response and duration of response, progression-free survival, and overall survival. The company plans to present data from the JULIET study in an upcoming medical meeting.
“We are encouraged by the FDA’s recognition in the potential of CTL019 for this indication, which follows our promising studies of this therapy for ALL and the FDA filing by Novartis in pediatric and young adult ALL that received priority review,” said the Penn team leader, Carl June, MD, director of the Center for Cellular Immunotherapies in the Perelman School of Medicine at the University of Pennsylvania. “Work with our collaborators at trial sites across the world is paving a path to bring personalized cell therapies to more patients with these devastating blood cancers.”