Reporter |
Editor |Xie Xin
China's First CAR-T Cell Therapy ProductAxicabtagene Ciloleucel Injection (Yikaida) Approved for Marketing This Month, on the evening of June 28, a pharmaceutical sales order for axicabtagene ciloleucel injection circulating in the industry showed that the retail price of the product was 1.2 million RMB per bag (approximately 68 mL).
Although this price has not been confirmed by Fosun Kite or Fosun Pharma, the "sky-high price" of RMB 1.2 million per bag immediately sparked heated discussion within the industry. This is because it may be the most expensive drug for a single treatment course ever launched in China.
So, is an anticancer drug priced at 1.2 million RMB per bag truly expensive, and is it worth it?
Yikaida is an anti-human CD19 CAR-T cell injection (YESCARTA) manufactured through technology transfer from Kite, a subsidiary of Gilead Sciences, and is planned for localized production in China. The indication applied for registration for this product is for the treatment of relapsed or refractory large B-cell lymphoma in adults (including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma (PMBCL), high-grade B-cell lymphoma, and DLBCL transformed from follicular lymphoma).
In fact, not only Yikaida, but all five CAR-T therapies currently on the market are "exorbitantly expensive." The U.S. Food and Drug Administration (FDA) has currently approved a total of five CAR-T therapies: Novartis's Kymriah, Gilead Kite's Yescarta and Tecartus, and Bristol Myers Squibb's Breyanzi and Abecma. These products are priced between $373,000 and $475,000 (equivalent to RMB 2.408 million to 3.066 million).
Although CAR-T therapy requires only a single patient infusion throughout the entire treatment course—meaning the drug is administered just once—the controversy over its pricing has never subsided since Novartis's Kymriah was launched in 2017.
CAR-T therapy is so expensive primarily because it is a personalized treatment modality. Given current technological constraints, the "drug" for each patient undergoing CAR-T therapy is unique.
The anti-tumor mechanism of CAR-T cell therapy involves harvesting autologous T cells from the patient and introducing genetic constructs that encode receptors recognizing tumor-specific antigens, along with gene sequences that facilitate T-cell activation, thereby generating CAR-T cells. T cells are naturally occurring immune effector cells in the human body capable of clearing non-self cells, including infected and tumor cells. The engineered CAR-T cells are equipped with both a tumor-targeting guidance system and an enhanced cytotoxic arsenal. Following ex vivo expansion and culture, these modified T cells are reinfused into the patient. Upon encountering tumor cells expressing the corresponding antigen, they become activated and undergo further proliferation, exerting potent and highly specific cytotoxic effects to eradicate the tumor.
Based on this principle, current cell therapy products must be "patient-specific". Even for the same target, differences in genetic design and manufacturing processes may lead to variations in quality control test items and standards, which cannot be directly copied or applied.
Furthermore, as a live cell therapeutic product, its manufacturing requires an exceptionally high level of environmental cleanliness. Each patient constitutes an independent batch, and every batch must undergo comprehensive testing. Only through rigorous manufacturing processes and stringent quality control can clinical safety and efficacy be maximized, alongside achieving a shorter manufacturing turnaround time and a higher production success rate. This is of critical significance for patients with relapsed or refractory diseases.
In terms of efficacy, previous registration studies of YESCARTA showed that the 1-year follow-up results for 101 adult patients with relapsed/refractory large B-cell lymphoma indicated: the best overall response rate was 82%, and the complete response rate reached 54%; at the 2-year follow-up: with a median follow-up of 27.1 months, 39% of subjects remained in response, of whom 37% remained in complete response. The 3-year follow-up results were announced in December 2019: with a median follow-up time of 39.1 months, the overall survival rate was 47%, and the median overall survival was 25.8 months.
Importantly, CAR-T therapy is generally used for last-line cancer patients, i.e., those who are likely already "out of treatment options."
However, as it is currently primarily indicated for late-line cancer patients, its current market potential is relatively limited, necessitating high pricing to achieve profitability. Furthermore, Yikaida is the first CAR-T therapy approved in China, leaving it with minimal market competition.
However, with the anticipated successive approvals of CAR-T therapies from JW Therapeutics, Legend Biotech, Novartis, and CARsgen Therapeutics, the prices of both Yescarta and other CAR-T therapies are expected to decline.