Gilead's Yescarta Receives FDA Orphan Drug Designation and Granted Priority Review in China

Yescarta (axicabtagene ciloleucel), the CD19 CAR-T cell therapy developed by Kite Pharma, a Gilead Sciences company, was recently granted two orphan drug designations (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of extranodal marginal zone lymphoma (extranodal MZL) and nodal marginal zone lymphoma (nodal MZL), respectively.

To date, Yescarta has been granted five Orphan Drug Designations (ODD) by the FDA, with the other three being for the treatment of follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL), and primary mediastinal large B-cell lymphoma (PMBCL).

Yescarta received approval from the U.S. FDA in October 2017, becoming the second CAR-T cell therapy approved for marketing globally, following Novartis’s Kymriah. The indications for Yescarta include the treatment of adult patients with relapsed or refractory large B-cell lymphoma (LBCL) who have previously undergone two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), high-grade B-cell lymphoma (HGBL), and DLBCL arising from follicular lymphoma (FL) (i.e., transformed FL, TFL). The indications for Kymriah include the treatment of acute lymphoblastic leukemia (ALL) in children and young adults (aged 2 to 25 years), as well as relapsed/refractory large B-cell lymphoma in adults (including DLBCL, transformed follicular lymphoma, and primary mediastinal B-cell lymphoma). According to the financial reports released by Gilead Sciences and Novartis, the global sales of Yescarta and Kymriah in 2019 were $456 million and $278 million, respectively.

In China, Yescarta (axicabtagene ciloleucel injection [proposed], code FKC876) is being developed by Fosun Kite Biotechnology Co., Ltd. (FOSUN Kite), a joint venture established by Shanghai Fosun Pharmaceutical Group and Kite Pharma. Earlier this month, Fosun Kite announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) had included the New Drug Application (NDA) for axicabtagene ciloleucel injection (proposed), a CAR-T cell therapy product, in the priority review program. The application is for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal B-cell lymphoma (PMBCL), high-grade B-cell lymphoma, and DLBCL transformed from follicular lymphoma.

Yiqililunsai Injection (code name FKC876) is a CD19-targeted autologous CAR-T cell therapy product that Fosun Kite has licensed from Kite Pharma for Yescarta (axicabtagene ciloleucel) technology and is authorized for localized production in China.

This product is the first CAR-T cell therapy product that Fosun Kite has advanced toward commercialization in China, and it is also the first CAR-T cell therapy product for which the National Medical Products Administration (NMPA) has formally accepted a marketing application to date. As a novel cancer treatment modality, FKC876 offers new hope and opportunities for patients in China with relapsed or refractory large B-cell lymphoma who have received two or more prior lines of systemic therapy.

Currently, CAR-T has become a hotspot in cancer immunotherapy. In addition to the two approved CAR-T cell therapies (Kymriah and Yescarta), several other CAR-T therapies are undergoing regulatory review, including Kite Pharma’s CD19 CAR-T cell therapy KTE-X19 (for the treatment of mantle cell lymphoma) and Bristol Myers Squibb’s CD19 CAR-T cell therapy lisocabtagene maraleucel (liso-cel, JCAR017, for the treatment of large cell lymphoma). Furthermore, a novel Fc-optimized immune-enhancing monoclonal antibody targeting CD19, tafasitamab, is also under regulatory review.

It is worth noting that the aforementioned four CD19 CAR-T therapies are all autologous CAR-T therapies, each requiring individualized manufacturing for every patient. The manufacturing process involves isolating the patient’s T cells, genetically modifying them ex vivo to express a chimeric antigen receptor (CAR) designed to target CD19, and then infusing the modified T cells back into the patient’s body, where they seek out and eliminate CD19-expressing cancer cells to exert their therapeutic effect.

Tafasitamab is an industrially manufactured, ready-to-use monoclonal antibody available for immediate administration. In terms of efficacy, tafasitamab is comparable to Kymriah and Yescarta. Regarding treatment costs, while both Kymriah and Yescarta are priced at hundreds of thousands of dollars, the cost of tafasitamab can be kept significantly lower. Some analysts have dubbed tafasitamab a “CAR-T cell therapy killer,” noting that its successful market launch would inevitably deliver a major blow to Kymriah and Yescarta.

Original Source: United StatesFDAOrphan Drug Database

Original Title:CD19 CAR-T Cell Therapy! Gilead’s Yescarta Granted Orphan Drug Designation by the U.S. FDA and Included in Priority Review in China!

Source: Bioon. For more information, please download the Bioon APP (http://www.bioon.com/m/)