On February 6, 2026, Kite, a subsidiary of Gilead Sciences, announced that the U.S. Food and Drug Administration (FDA) had approved an update to the prescribing information for Yescarta (axicabtagene ciloleucel), removing the previous usage restrictions for patients with relapsed/refractory (R/R) primary central nervous system lymphoma (PCNSL).

About the Label Update

This label update further validates the reliable safety profile of Yescarta in eligible patients with relapsed/refractory primary central nervous system lymphoma. Yescarta is also the only CAR-T cell therapy approved for relapsed/refractory large B-cell lymphoma from which the aforementioned usage restrictions have been removed.

Primary central nervous system lymphoma is a rare, rapidly progressive lymphoma originating in the brain, spinal cord, eyes, or cerebrospinal fluid. The disease has an extremely poor prognosis, with a 5-year survival rate of approximately 30%. More than half of patients experience disease relapse after first-line treatment, with a median overall survival of only about two months following relapse, underscoring the urgent clinical demand for novel and optimized treatment regimens.

The FDA’s approval is based on positive results from an investigator-initiated Phase 1 clinical trial conducted at the Dana-Farber Cancer Institute, which enrolled patients with relapsed/refractory primary central nervous system lymphoma.

About Central Nervous System Lymphoma

Central nervous system lymphoma (CNSL) is an aggressive, rare form of non-Hodgkin lymphoma that can arise primarily (primary CNSL) or metastasize to (secondary CNSL) the brain, eyes, spinal cord, or cerebrospinal fluid.

Approximately 1,500 new cases of primary central nervous system lymphoma are diagnosed in the U.S. annually, accounting for 3% of all primary brain tumors and 1% of all non-Hodgkin lymphomas. Historically, the disease carries an extremely poor prognosis with a 5-year survival rate of only 30%, and it predominantly affects the elderly and immunocompromised individuals. There is currently no standard treatment for relapsed/refractory central nervous system lymphoma, representing an area of high unmet medical need.

About the Study

This Phase 1 safety study enrolled a total of 18 patients (13 with primary central nervous system lymphoma and 5 with secondary central nervous system lymphoma), with the first 6 patients used to assess treatment-limiting toxicities (TLT).

The primary endpoint was safety, evaluated by the incidence of treatment-limiting toxicities and the rate of Grade ≥3 adverse events (AEs).

Secondary endpoints included objective response rate (ORR), complete response rate (CRR), duration of response (DOR), progression-free survival (PFS), and overall survival (OS).

About Yescarta (Axicabtagene Ciloleucel)

Yescarta is a CD19-directed genetically modified autologous T-cell immunotherapy approved for the treatment of:

Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or relapses within 12 months of first-line chemoimmunotherapy.

Adult patients with relapsed/refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified (DLBCL-NOS), primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

Adult patients with relapsed/refractory follicular lymphoma (FL) after two or more lines of systemic therapy.

This indication is approved under the accelerated approval pathway based on response rates. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory clinical trials.