Policy & Regulation
Daiichi Sankyo Submits Application for CAR T Therapy Axicabtagene Ciloleucel for Treatment of Patients with Certain Relapsed/Refractory B-cell Lymphomas in Japan
2 April 2020 - - Japan-based pharmaceutical company Daiichi Sankyo Company, Ltd. has submitted a New Drug Application to Japan's Ministry of Health, Labour and Welfare for chimeric antigen receptor T cell therapy axicabtagene ciloleucel for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma and related lymphomas, the company said.

In January 2017, Daiichi Sankyo received exclusive development, manufacturing and commercialisation rights for axicabtagene ciloleucel in Japan from California-based Kite, a Gilead company.

The Japan NDA submission is based on previous pivotal trial data conducted globally by Kite for axicabtagene ciloleucel in addition to results from a phase 2 bridging study conducted by Daiichi Sankyo in Japan.

Both trials included patients with four aggressive types of relapsed/refractory B-cell lymphomas including diffuse large B cell lymphoma (DLBCL); primary mediastinal B-cell lymphoma (PMBCL); transformed follicular lymphoma ; and high-grade B cell lymphoma.

The Japanese trial met its primary endpoint for objective response rate, and data will be presented at an upcoming medical meeting.

Axicabtagene ciloleucel is a CAR T cell therapy directed against CD19 (a cell membrane protein), which harnesses a patient's own immune system to fight B-cell lymphoma. Axicabtagene ciloleucel received Orphan Drug Designation from the Japan Ministry of Health, Labour and Welfare in 2018 for the treatment of DLBCL, PMBCL, TFL and high-grade B-cell lymphoma.

Axicabtagene ciloleucel is approved in the US and Europe for patients with certain types of relapsed or refractory B-cell lymphoma based on the results of the pivotal ZUMA-1 study.

There were an estimated 509,000 new cases and about 248,000 deaths globally from non-Hodgkin lymphoma in 2018.1 In Japan, there were nearly 21,000 new cases of NHL reported in 2012.2

About 85% of NHLs are B-cell lymphomas.

DLBCL is the most commonly diagnosed NHL, representing about one in five cases.

Treatment advances have led to improved outcomes for patients with certain types of NHL, but relapsed or refractory disease has remained a significant treatment challenge.

DLBCL is considered an aggressive form of the disease; however, the majority of DLBCL patients do achieve complete and sustained remission on initial treatment (chemotherapy plus targeted therapy).

Approximately 40% of patients experience relapse or resistance and may subsequently receive additional chemotherapy and/or stem cell transplant if eligible; those who are ineligible for ASCT or who relapse after transplantation have a poor prognosis.

New and novel treatments such as CAR T cell therapies are providing additional options for some patients with relapsed/refractory DLBCL after two or more lines of treatments have been tried.6

The phase 2 multicenter, open-label, single-arm study was planned to evaluate efficacy and safety of axicabtagene ciloleucel in Japanese patients with several aggressive types of large B-cell lymphoma that is refractory or relapsed following one or more lines of standard treatment including drug therapy or stem cell transplant.

The study enrolled patients with DLBCL, PMBCL, TFL and high-grade B cell lymphoma.

The primary efficacy endpoint is investigator-assessed objective response rate. Secondary efficacy endpoints include centrally evaluated ORR, duration of response, progression-free survival and overall survival. The study will also measure safety and pharmacokinetics.

Enrollment has completed and the study is ongoing at several institutions in Japan.
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