The FDA accepted the biologics license application (BLA) and granted priority review to KTE-X19, an investigational CAR T-cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL), according to Kite, the agent’s developer.
The BLA is supported by data from the ongoing phase II ZUMA-2 trial, which found that 93% of patients in the study responded to a single infusion of KTE-X19, including 67% of patients achieving a complete response, after a median follow-up of 12.3 months.
Findings from this trial were presented at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition, held in in Orlando, Florida, from December 7-10, 2019.
“Despite recent advances, patients with relapsed/refractory mantle cell lymphoma currently face a significant lack of effective options once their disease no longer responds to currently available therapy,” Ken Takeshita, MD, global head of clinical development for Kite, said in a press release. “Based on the encouraging results for KTE-X10, we are eager to continue discussions with the FDA on how to bring this innovative treatment to these patients who may benefit from CAR T therapy.”
In the single-arm, multicenter, open-label study, which enrolled 74 leukapharesed adult patients (≥18 years old) with MCL whose disease is refractory to or has relapsed following up to 5 prior lines of therapy, researchers looked to evaluate the efficacy and safety after a single infusion of KTE-X19. The primary endpoint is the objective response rate, defined as the combined rate of complete responses and partial responses as assessed by an Independent Radiologic Review Committee.
Secondary endpoints included duration of response, progression-free survival, overall survival, incidence of adverse events, incidence of anti-CD19 CAR antibodies, levels of anti-CD19 CAR T cells in the blood, levels of cytokines in serum, and changes over time in the EQ-5D scale score and visual analogue scale score.
In the safety analysis, grade 3 or higher cytokine release syndrome (CRS) and neurologic events were observed in 15% and 31% of patients, respectively. No grade 5 CRS or neurologic events have occurred this far.
KTE-X19 is still investigational and not yet approved in any country globally.
The Prescription Drug User Fee Act (PDUFA), or target action date, is August 10, 2020. Recently, the European Medicines Agency (EMA) validated the Marketing Authorization Application for KTE-X19 in the European Union. Additionally, the agent has already been granted breakthrough therapy designation by the FDA and Priority Medicines (PRIME) designation by the EMA for relapsed or refractory MCL.
U.S. FDA Grants Priority Review for Kite’s KTE-X19 Biologics License Application (BLA) in Relapsed or Refractory Mantle Cell Lymphoma [news release]. Santa Monica, California. Published February 10, 2020. investors.gilead.com/news-releases/news-release-details/us-fda-grants-priority-review-kites-kte-x19-biologics-license. Accessed February 10, 2020.