August 2021: The FDA has given axicabtagene ciloleucel (Yescarta, Kite Pharma, Inc.) expedited approval for adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.
A single-arm, open-label, multicenter trial (ZUMA-5; NCT03105336) evaluated axicabtagene ciloleucel, a CD19-directed chimeric antigen receptor (CAR) T cell therapy, in adult patients with relapsed or refractory FL after two or more lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent, in adult patients with relapsed A single intravenous infusion of axicabtagene ciloleucel was given after lymphodepleting chemotherapy.
An impartial review committee defined the major efficacy measures: objective response rate (ORR) and duration of response (DOR). The ORR was 91 percent (95 percent CI: 83, 96) among 81 patients in the primary efficacy analysis, with a complete remission (CR) rate of 60 percent and a median time-to-response of one month. The median DOR was not reached, and 76.2 percent of patients remained in remission after one year (95 percent CI: 63.9, 84.7). The ORR was 89 percent (95 percent CI: 83, 94) for all leukapheresed patients in this trial (n=123), with a CR rate of 62 percent.
A boxed warning for cytokine release syndrome (CRS) and neurologic toxicities is included in the prescribing material for axicabtagene ciloleucel. CRS occurred in 88 percent (Grade 3, 10%) of patients with non-lymphoma Hodgkin’s (NHL) in investigations using axicabtagene ciloleucel, while neurologic toxicities occurred in 81 percent (Grade 3, 26 percent). CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, febrile neutropenia, nausea, infections with pathogen unspecified, decreased appetite, chills, diarrhoea, tremor, musculoskeletal pain, cough, hypoxia, constipation, vomiting, arrhythmias, and dizziness are the most common non-laboratory adverse reactions (incidence 20%) in patients with NHL.
Reference : https://www.fda.gov/
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