Yescarta was awarded an orphan drug for four types of lymphoma

Daiichi Sankyo, a Japanese pharmaceutical company, recently announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has awarded CAR-T cell therapy axicabtagene ciloleucel (formerly known as KTE-C19) to treat diffuse large B-cell lymphoma (D LBCL), primary vertical (thymus) large B-cell lymphoma (PMBCL), high-level B-cell lymphoma (HGBL), transformational fiery lymphoma (TFL) orphan drug eligibility, the above four types of lymphoma are invasive non-Hodgkin lymphoma (NHL).
is a drug used to prevent, treat and diagnose rare diseases, which are commonly referred to as "orphan diseases", a category of diseases with very low incidence. According to the World Health Organization (WHO), diseases that affect 0.65 to 1 per 1,000 people in the total population can be defined as rare diseases. However, according to the specific situation of their own countries, there are some differences in the criteria for the identification of rare diseases. In Japan, rare diseases are defined as the type of disease that affects fewer than 50,000 people. Drugs that are eligible for orphan drugs are eligible for incentives for research and development, including guidance and subsidies for clinical development activities, priority consultation and priority review of clinical development.Dr. Kooichi Akahane, Head of Oncology,
's First Third Pharmaceutical Research and Development Division, said MHLW's granting of the axicabtagene ciloleucel orphan drug is an important step in accelerating the clinical development of the product in Japan and highlights the significant unsecured medical needs of these groups of patients with invasive or resusable B-cell lymphoma. The orphan drug qualification also represents the third orphan drug qualification obtained by the first three pharmaceutical oncology pipeline assets. We look forward to working with Japanese regulators to bring this important cell therapy to Japanese patients as soon as possible.
, developed by Gilead-owned company Kite Pharma, was granted exclusive rights to the development, manufacture and commercialization of the CAR-T therapy in Japan in January 2017.
, two CAR-T therapies have been approved for sale: Novart's Kymriah (Tisagenlecleucel, CTL019) and Gilead's Yescarta (axicabtagene ciloleucel, KTE-C19). CAR-T therapy, unlike conventional small molecules or biological therapies, is a live T-cell therapy product. Both Kymriah and Yescarta are based on the gene modification of a patient's T-cells to express an inset antigen -CAR designed to target antigen CD19, an antigen protein expressed on the surface of a variety of blood tumor cells, including B-cell lymphoma and leukemia cells.
U.S. market, Kymriah and Yescarta were approved in August and October 2017, respectively. To date, Kymriah has approved the symptoms as: (1) for refractic or relapsed at least 2 times in B-ALL children and young adult patients; Yescarta has approved the treatment of adult patients with recurring or refractic large B-cell lymphoma (LBCL) who have received two or more systemic therapies, including DLBCL, PMBCL, high-level B-cell lymphoma (HGBL), and DLBCL (i.e., transformative FL, TFL) derived from folytic lymphoma (FL). (Bio Valley)