The U.S. Food and Drug Administration (FDA) granted priority review to CTL019 (tisagenlecleucel-T), an investigational chimeric antigen receptor (CAR) T-cell therapy, for the treatment of relapsed/refractory B-cell acute lymphocytic leukemia (ALL) in pediatric and young adult patients. CTL019 was submitted for review through the Biologics License Application process.
This decision was based on results from the ELIANA study, a single arm, open-label, multi-center, phase II study, that was conducted globally across 25 centers. Of the 50 enrolled patients, 82 percent (n=41) achieved either complete remission (CR) or CR with incomplete blood count recovery three months after treatment with CTL019.
Grade 3 or 4 cytokine release syndrome occurred in 24 patients, but no deaths from adverse events (AE) were reported. Another 15 percent of patients suffered neurologic and psychiatric AEs such as confusion, delirium, encephalopathy, agitation, and seizure.
The FDA previously granted CTL019 breakthrough therapy designation for the treatment of patients with relapsed/refractory ALL.
Source: Novartis press release, March 29, 2017.