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FDA grants priority review to CTL019 for ALL
The FDA granted priority review to tisagenlecleucel-T, an investigative chimeric antigen receptor T-cell therapy intended for children and young adults with relapsed and refractory B-cell acute lymphoblastic leukemia.
The FDA based the priority review designation of tisagenlecleucel-T (CTL019, Novartis) on results of the phase 2 ELIANA study, a global chimeric antigen receptor (CAR) T-cell trial that included 50 patients from 25 centers in the United States, Europe, Canada, Japan and Australia.
Results — presented in December at the ASH Annual Meeting and Exposition — showed 41 (82%) patients infused with CAR T cells achieved complete remission or complete remission with incomplete blood count recovery by 3 months after CTL019 infusion.
Forty-eight percent of patients in the trial experienced grade 3 or grade 4 cytokine release syndrome, but researchers reported no deaths due to the complication. Fifteen percent of patients experienced grade 3 neurological and psychiatric events, including delirium, confusion, agitation, seizure and encephalopathy.
Investigators reported no cerebral edema, and no grade 4 neurological or psychiatric events.
A separate multicenter trial and single-site trial led by Children’s Hospital of Pennsylvania evaluated the efficacy and safety of CL019 among children and young adults with relapsed/refractory B-cell ALL.
“Even if a patient has difficult-to-treat relapsed/refractory leukemia, we have seen treatment with CTL019 in clinical trials put cancer into remission,” lead investigator Stephan Grupp, MD, PhD, director of the cancer immunotherapy frontier program and director of translational research for the Center for Childhood Cancer Research at CHOP, said in the press release. “This could be a first-of-its-kind treatment with exciting potential to help pediatric and young adult relapsed/refractory B-cell ALL patients.”
The University of Pennsylvania developed CTL019 in 2012. Novartis and the university entered into a global collaboration in 2012 to research, develop and commercialize CAR T-cell therapies for treatment of cancer.
“The past five years have seen tremendous progress in the development and application of cellular engineering in an effort to personalize the treatment of cancer,” Carl June MD, director of the Center for Cellular Immunotherapies in Perelman School of Medicine at University of Pennsylvania, said in a Novartis-issued press release. “We now know that it is possible to treat patients in clinical trials across the world using this approach, and the results we have observed mark a potential new paradigm in the treatment of blood cancers that have not responded to standard therapies.”
Novartis intends to submit another biologics license application with the FDA later this year for use of CTL019 to treat adults with relapsed/refractory diffuse large B-cell lymphoma.