FDA approves label update for axi-cel for the treatment of R/R PCNSL

On February 6, 2026, the U.S. Food and Drug Administration (FDA) approved an update to the prescribing information (PI) for axicabtagene ciloleucel (axi-cel), a CD19-directed chimeric antigen receptor (CAR) T-cell therapy, removing the previous limitations of use in patients with relapsed or refractory (R/R) primary central nervous system lymphoma (PCNSL).¹  

This update was based on positive results from an investigator-sponsored phase I study, conducted by the Dana-Farber Cancer Institute, investigating the safety and efficacy of axi-cel in patients with PCNSL (n = 13) or secondary central nervous system lymphoma (SCNSL; n = 5).¹ The primary endpoint was safety, measured by treatment-limiting toxicity (TLT) rate and the rate of Grade ≥3 adverse events (AEs). Secondary endpoints included overall response rate (ORR), complete response rate (CRR), duration of response (DoR), progression-free survival (PFS), and overall survival (OS). 

In this study, 85% of patients with R/R PCNSL experienced neurologic toxicities, with Grade 3 events occurring in 31% of these patients.¹ The Grade 3/4 AEs recorded were hypotension (23%), encephalopathy (15%), seizure (15%), gait disturbance (8%), headache (8%), hypoxia (8%), muscular weakness (8%), nausea (8%), pyrexia (8%), thrombosis (8%), and tremor (8%).¹  

This update is expected to expand access to axi-cel as a potential therapy for patients with R/R PCNSL, who have historically had limited treatment options.¹