February 6, 2026 — Leads & Copy —
Kite, a Gilead Company, announced that the U.S. Food and Drug Administration (FDA) has approved an update to the Yescarta® (axicabtagene ciloleucel) prescribing information, removing the previous Limitations of Use in patients with relapsed or refractory (R/R) primary central nervous system lymphoma (PCNSL).
The updated label reinforces the safety data of Yescarta in eligible patients with R/R PCNSL. Yescarta is the only CAR T-cell therapy approved for R/R large B-cell lymphoma to have this Limitations of Use removed.
Primary central nervous system lymphoma is a rare and fast‑growing lymphoma that originates in the brain, spinal cord, eye, or cerebrospinal fluid. The prognosis for PCNSL remains poor, with a five‑year survival rate of approximately 30%. More than half of patients see their disease return after the first treatment, with subsequent survival of approximately two months, highlighting the need for better treatment options.
The FDA decision is based on positive results from a Phase 1 investigator-sponsored study conducted by Dana-Farber Cancer Institute, which included patients with R/R PCNSL.
According to Dr. Lakshmi Nayak, Director of the Center for CNS Lymphoma, Dana-Farber Cancer Institute and Associate Professor of Neurology, Harvard Medical School, their study highlighted the safety of axi-cel in central nervous system lymphoma and supported the FDA’s decision. She added that the update to the axi-cel prescribing information provides clinicians with important evidence for patients who have historically had limited treatment options.
In the Phase 1 study, neurologic toxicities occurred in 85% (11/13) of patients with R/R PCNSL. Thirty-one percent (4/13) of patients had Grade 3 neurologic toxicities. The Grade 3 or 4 adverse events were hypotension (23%; 3/13), encephalopathy (15%; 2/13), seizure (15%; 2/13), gait disturbance (8%; 1/13), headache (8%; 1/13), hypoxia (8%; 1/13), muscular weakness (8%; 1/13), nausea (8%; 1/13), pyrexia (8%; 1/13), thrombosis (8%; 1/13), and tremor (8%; 1/13).
Dr. Gallia Levy, Senior Vice President and Global Head of Development, Kite, said that they are encouraged by the positive results of the safety study in patients with central nervous system lymphoma, who were previously excluded from the trials supporting Yescarta’s approval. Levy added that they appreciate the FDA’s timely review and decision, which expands access to Yescarta for patients with primary central nervous system lymphoma, and are grateful to the patients and clinicians who made this progress possible.
Central nervous system lymphoma (CNSL) is an aggressive and rare form of non-Hodgkin lymphoma that has either originated in (primary) or spread (secondary) to the brain, eye, spinal cord, or cerebrospinal fluid. There is an estimated annual incidence of 1,500 cases of PCNSL in the United States, comprising 3% of all primary brain tumors and 1% of all cases of non-Hodgkin lymphoma. Its prognosis has historically been poor, with a five-year survival rate of only 30%. CNSL is most likely to be seen in the elderly and people with a compromised immune system. R/R CNSL is considered an area of unmet clinical need with no standard-of-care treatment options.
The Phase 1 safety study enrolled 18 patients (13 PCNSL, 5 SCNSL), of whom the first six patients were observed for treatment-limiting toxicities (TLTs). The primary endpoint was safety, measured by rate of TLTs and ≥ Grade 3 adverse events (AEs). Secondary endpoints included objective response rate, complete response rate, duration of response, progression-free survival and overall survival (OS).
YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy; adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma; and adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.
Please see full Prescribing Information, including BOXED WARNING below and Medication Guide. Please see full Prescribing Information, including BOXED WARNING and Medication Guide.
Gilead and Kite Oncology are working to transform how cancer is treated through next-generation therapies, combinations and technologies. The oncology portfolio and pipeline address the greatest gaps in care, from antibody-drug conjugate technologies and small molecules to cell therapy-based approaches.
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Source: Kite
