At a Glance
FDA removed the prior PCNSL use limitation from Gilead’s Yescarta label.
The update does not add PCNSL as a separate approved indication.
Supported by safety results from the investigator-sponsored study NCT04608487.
Clinicians can now use Yescarta in eligible PCNSL patients without label restrictions.
Written By: Nikita Chaudhari, BPharm
Reviewed By: Pharmacally Editorial Team
Kite, a company of Gilead Sciences announced that the U.S. Food and Drug Administration has approved an update to the prescribing information for Yescarta (axicabtagene ciloleucel). The FDA has removed the previous “Limitations of Use” for patients with relapsed or refractory primary central nervous system lymphoma (PCNSL).
With this update, Yescarta becomes the only CAR T-cell therapy approved for relapsed or refractory large B-cell lymphoma to no longer carry a limitation related to PCNSL. The revised label reflects accumulating safety data supporting its use in eligible patients with disease involvement in the central nervous system.
Addressing a high-risk lymphoma with limited options
Primary central nervous system lymphoma is a rare, fast-growing form of non-Hodgkin lymphoma that arises in the brain, spinal cord, eyes, or cerebrospinal fluid. Despite aggressive treatment, long-term outcomes remain poor. About half of patients relapse after initial therapy, and survival after recurrence can be measured in months, underscoring the lack of effective options in this setting.
Study data behind the FDA decision
The FDA’s decision was supported by positive safety findings from a Phase 1 investigator-sponsored study (NCT04608487) conducted at Dana-Farber Cancer Institute. The study enrolled patients with relapsed or refractory PCNSL, a population historically excluded from pivotal CAR T-cell trials.
“We are pleased that our study, which highlighted the safety of axi-cel in central nervous system lymphoma, supported the FDA’s decision,” said Lakshmi Nayak, MD, Director of the Center for CNS Lymphoma at Dana-Farber and Associate Professor of Neurology at Harvard Medical School. “This update provides clinicians with meaningful evidence for patients who have long had very limited treatment options.”
Safety profile observed in the Phase 1 trial
The primary endpoint of the study was safety, assessed by rates of treatment-limiting toxicities and Grade 3 or higher adverse events. Secondary endpoints included objective response rate, duration of response, progression-free survival, and overall survival.
Among the 13 patients with relapsed or refractory primary CNS lymphoma treated in the study, neurologic toxicities were reported in 85 percent of patients, with Grade 3 neurologic events observed in 31 percent. Reported Grade 3 or 4 adverse events included hypotension, encephalopathy, seizure, hypoxia, thrombosis, and other neurologic and systemic events, each occurring in a minority of patients.
These toxicities were consistent with the known safety profile of axicabtagene ciloleucel in systemic large B-cell lymphoma and did not reveal new or unexpected central nervous system–specific risks. The study did not exceed predefined thresholds for treatment-limiting toxicities, indicating that adverse events were manageable with established CAR T-cell safety protocols in this high-risk population.
Expanding access to CAR T-cell therapy
“We are encouraged by the safety results in patients with central nervous system lymphoma, who were previously excluded from trials supporting Yescarta’s approval,” said Gallia Levy, MD, PhD, Senior Vice President and Global Head of Development at Kite. “We appreciate the FDA’s timely review, which helps expand access to Yescarta for patients with one of the most aggressive and underserved forms of lymphoma.”
About Yescarta
Yescarta is a CD19-directed, genetically modified autologous T-cell immunotherapy approved for multiple forms of large B-cell lymphoma, including disease that is refractory to first-line chemoimmunotherapy or relapses within 12 months, as well as relapsed or refractory disease after two or more lines of systemic therapy. It is also approved for relapsed or refractory follicular lymphoma under accelerated approval.
For complete details on indications, adverse reactions, boxed warnings, and precautions, readers should consult the full Yescarta Prescribing Information available from Kite/Gilead.
References
FDA Approves Label Update for Kite’s Yescarta® for Relapsed/Refractory Primary Central Nervous System Lymphoma, 06 February 2026, https://www.gilead.com/news/news-details/2026/fda-approves-label-update-for-kites-yescarta-for-relapsedrefractory-primary-central-nervous-system-lymphoma
Axi-cel in CNS Lymphoma, ClinicalTrials.gov ID NCT04608487, https://clinicaltrials.gov/study/NCT04608487
Karschnia, P., Dietrich, J. Neurological complications of CAR T cell therapy for cancers. Nat Rev Neurol 21, 422–431 (2025). https://doi.org/10.1038/s41582-025-01112-8
Yescarta Prescribing Information, https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.kitepharma.com%2F-%2Fmedia%2Ffiles%2Fpdfs%2Fmedicines%2Foncology%2Fyescarta%2Fyescarta-pi.pdf&esheet=54418368&newsitemid=20260205203420&lan=en-US&anchor=Prescribing+Information&index=1&md5=06e5bdd229ea17e6abd286400d21722f