SANTA MONICA, Calif. — Kite, a Gilead Company, said the U.S. Food and Drug Administration has approved an update to the prescribing information for Yescarta (axicabtagene ciloleucel), removing the previous Limitations of Use for patients with relapsed or refractory primary central nervous system lymphoma.
The company said the updated label reinforces the safety profile of Yescarta in eligible patients with relapsed or refractory PCNSL and makes it the only CAR T-cell therapy approved for relapsed or refractory large B-cell lymphoma to have this limitation removed.
Primary central nervous system lymphoma is a rare and aggressive form of non-Hodgkin lymphoma that originates in the brain, spinal cord, eye, or cerebrospinal fluid. Prognosis remains poor, with an estimated five-year survival rate of about 30%. More than half of patients experience disease relapse after initial treatment, and subsequent survival can be as short as two months, underscoring the need for additional treatment options.
The FDA’s decision was based on positive results from a Phase 1 investigator-sponsored study conducted by Dana-Farber Cancer Institute that included patients with relapsed or refractory PCNSL.
“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 Cancer Institute and Associate Professor of Neurology at Harvard Medical School. “This update to the axi-cel prescribing information provides clinicians with important evidence for patients who have historically had very limited treatment options.”
In the Phase 1 study, neurologic toxicities were observed in 85% (11 of 13) of patients with relapsed or refractory PCNSL, with 31% (4 of 13) experiencing Grade 3 neurologic toxicities. Grade 3 or 4 adverse events included hypotension (23%; 3 of 13), encephalopathy (15%; 2 of 13), seizure (15%; 2 of 13), gait disturbance (8%; 1 of 13), headache (8%; 1 of 13), hypoxia (8%; 1 of 13), muscular weakness (8%; 1 of 13), nausea (8%; 1 of 13), pyrexia (8%; 1 of 13), thrombosis (8%; 1 of 13), and tremor (8%; 1 of 13).
“We 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,” said Gallia Levy, MD, PhD, Senior Vice President and Global Head of Development at Kite. “We appreciate the FDA’s timely review and decision, which expands access to Yescarta for patients with primary central nervous system lymphoma—one of the most aggressive and underserved forms of the disease—and we are deeply grateful to the patients and clinicians who made this progress possible.”
