#Japan #Tokyo #Gilead Sciences #Kite Pharma #Brexucabtagene

Brexucabtagene Autoleucel's New Status

On January 16th, Gilead Sciences, headquartered in Chiyoda, Tokyo, announced that the Japanese Ministry of Health, Labour and Welfare has designated Brexucabtagene autoleucel as an orphan drug for mantle cell lymphoma (MCL) and B-cell acute lymphoblastic leukemia (B-ALL). This designation highlights both the rarity of the conditions and the critical need for effective treatments.

Orphan drug status is given to treatments that target diseases affecting fewer than 50,000 patients in Japan, indicating a high medical necessity. MCL is classified as a type of non-Hodgkin lymphoma and constitutes approximately 3% of malignant lymphomas in Japan. According to a 2023 survey by the Ministry of Health, around 2,000 individuals suffer from MCL. Unfortunately, many patients experience recurrent relapses and face severe challenges, with typical survival rates ranging from 5 to 7 years.

Due to the lack of established standard treatments available for MCL, the outlook remains poor, particularly as the number of treatment lines increases. Thus, novel treatment options like Brexucabtagene autoleucel are highly anticipated.

B-ALL is characterized by the excessive growth of immature lymphocytes in the blood, bone marrow, and other organs, arising from B-cell or T-cell lineages. The 2023 survey indicated about 9,000 reported cases of ALL in Japan, where 5-10% of patients show resistance to initial treatment, and 30-60% experience relapse. The five-year survival rate for relapsed patients is reported to be below 10%. Especially among adults, as treatment regimens intensify, the overall complete response rate declines, creating a dire need for alternative therapeutic strategies. B-ALL accounts for roughly 75% of all ALL cases.

Brexucabtagene autoleucel is an innovative CAR T-cell therapy targeting the CD19 antigen, previously deemed a groundbreaking treatment based on the ZUMA-2 and ZUMA-3 trials conducted by Kite Pharma, a Gilead company. It was first approved as a CAR T-cell therapy for relapsed and refractory MCL in the U.S. and Europe back in 2020, and as of now, it has been recognized in 40 countries and regions. Similarly, the treatment for adult patients with relapsed and refractory B-ALL gained approvals in the U.S. in 2021 and in Europe in 2022, with adoption in 39 countries to date.

About Kite Pharma

Kite, located in Santa Monica, California, is at the forefront of biopharmaceuticals and a pioneer in cell therapy aimed at curing and treating cancer. With a large-scale manufacturing network for CAR T-cell therapy, it provides solutions that are transforming cancer treatment for countless patients. For further information, visit www.kitepharma.com.

About Gilead Sciences

Gilead Sciences is dedicated to advancing progress in healthcare for over three decades, striving to create a healthier world. Focusing on innovative solutions for life-threatening diseases including HIV, viral hepatitis, COVID-19, and cancer, Gilead operates in more than 35 countries, headquartered in Foster City, California.