Kite, a Gilead Company, announced the FDA 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 robust 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. Prognosis for PCNSL remains poor, with a five‑year survival rate of approximately 30%. More than half of patients see their disease come back after the first treatment, with subsequent survival of approximately two months, highlighting the urgent need for new and 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.
“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, Dana-Farber Cancer Institute and Associate Professor of Neurology, 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 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).
“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, 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.”
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