Kite Pharma said Monday that it submitted a filing to the European Medicines Agency seeking approval of axicabtagene ciloleucel, which the company noted represents the first CAR T-cell therapy submitted to the regulator. The application seeks clearance for patients with relapsed/refractory diffuse large B-cell lymphoma, transformed follicular lymphoma and primary mediastinal B-cell lymphoma who are ineligible for autologous stem-cell transplant.
Kite explained that its submission was supported by data from the ZUMA-1 trial of 101 patients assigned to treatment with a single infusion of axicabtagene ciloleucel. Results, which were unveiled in February, demonstrated that the overall response rate was 82 percent after a single infusion of the therapy, while 44 percent of patients remained in response after a median follow-up of 8.7 months. Kite said the most common severe side effects included cytokine release syndrome and neurologic events, which were "generally reversible." The company also noted when the ZUMA-1 results were released that there were no cases of cerebral oedema.
CEO Arie Belldegrun suggested the European filing "marks an important global milestone in the development of engineered T-cell therapy," adding that Kite would be working with regulators "to help bring this potentially transformative therapy to patients in the EU."
Axicabtagene ciloleucel, previously known as KTE-C19, was granted orphan drug status by European regulators in 2015, and later received access to the EMA's Priority Medicines initiative. A marketing application for the therapy, seeking approval in refractory aggressive non-Hodgkin lymphoma, has also been awarded priority review by the FDA, with a target action date of November 29.
Meanwhile, an FDA advisory panel backed approval of Novartis' investigational CAR-T therapy tisagenlecleucel-T earlier in July for patients aged three to 25 years with relapsed/refractory B-cell acute lymphoblastic leukaemia (ALL). In March, Juno Therapeutics ended development of its CAR-T drug JCAR015 in adults with relapsed or refractory ALL amid safety issues in the Phase II ROCKET trial, including five patient deaths due to cerebral oedema.
For related analysis, see ViewPoints: The data's all here for Kite and Novartis, but will there be a failure to launch? See also Analyst Notes: Success of anti-CD19 CAR-T therapies hinges on long-term durability and safety.
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