The US Food and Drug Administration (FDA) approved tisagenlecleucel for the treatment of pediatric and young adult patients up to 25 years of age with refractory or in second or later relapse B-cell precursor acute lymphoblastic leukemia (ALL).¹

This approval marks the first chimeric antigen receptor T cell (CAR-T) therapy, which utilizes the patients’ own genetically-modified T-cells to target the CD19 antigen on the surface of leukemia cells. Once modified, the cells are injected back into the patient.

The FDA based its approval on data collected from the phase 2 ELIANA (ClinicalTrials.gov Identifier: NCT02435849) trial that assessed the outcomes of 63 evaluable pediatric and young adult patients with relapsed or refractory B-cell precursor ALL.

Results showed that 83% of patients (95% CI, 71%-91%) achieved complete response (CR) or CR with incomplete blood count recovery (CRi) within 3 months of treatment. There was also no evidence of minimal residual disease (MRD) ― a marker prognostic for potential relapse ― in patients. A median duration of remission was not reached (95% CI, 7.5 months-not evaluable).

The most frequently reported grade 3 or 4 adverse events (AE) cytokine release syndrome and neurologic events. Other commonly occurring AEs include serious infections, hypotension, fever, hypoxia, and acute kidney injury.

Reference

1. FDA approval brings first gene therapy to the United States [news release]. Silver Spring, MD: US Food and Drug Administration. Updated August 30, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm. Accessed August 30, 2017.