NEW YORK – The US Food and Drug Administration on Friday granted accelerated approval to the autologous CAR T-cell therapy axicabtagene ciloleucel (Gilead's Yescarta) as a treatment for relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.
The FDA based the accelerated approval on the results of the Phase II ZUMA-5 clinical trial, which evaluated the treatment across relapsed or refractory patients with subtypes of indolent non-Hodgkin lymphoma, including follicular lymphoma and marginal zone lymphoma. Of the 81 patients with follicular lymphoma who were evaluable for the trial's efficacy analysis, 91 percent responded to axicabtagene ciloleucel, and an estimated 74 percent of patients remained in complete remission after 18 months. After 14.5 months, the median duration of response was not yet reached. In an initial safety analysis of 146 patients, eight percent of patients experienced grade 3 or higher cytokine release syndrome and 21 percent experienced grade 3 or higher neurologic toxicities.
Axicabtagene ciloleucel, made by Santa Monica, California-based Kite Pharma, a Gilead subsidiary, is already approved for relapsed or refractory large B-cell lymphomas, and this expansion of the treatment's label marks the first CAR T-cell therapy approved for patients with indolent follicular lymphoma.
Kite noted that the prescribing information for Yescarta includes a boxed warning for risk of cytokine release syndrome and neurological toxicities, and the drug has been approved with a risk evaluation and mitigation strategy for these risks.