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[Asia Economy Reporter Lee Chun-hee] Ultra-high-priced therapies such as the chimeric antigen receptor (CAR)-T cell therapy 'Kymriah' (360 million KRW) and the gene therapy 'Zolgensma' (approximately 2.5 billion KRW) are being introduced consecutively in South Korea. Both therapies are gaining attention as 'one-shot' treatments that can cure rare diseases?acute lymphoblastic leukemia and spinal muscular atrophy (SMA), respectively?with a single injection, leading to either the application or expected application of National Health Insurance coverage.

Although these treatments are extremely expensive, the cost significantly decreases when covered by health insurance. In the case of Kymriah, while the price exceeds 500 million KRW in the United States, where private insurance predominates, the reimbursement ceiling in South Korea is set at 360 million KRW. If National Health Insurance is applied once per patient for a lifetime, the price drops sharply to around 6 million KRW. Zolgensma's price, which reaches 2.5 billion KRW, is also expected to decrease substantially. However, since this is not a reduction in the drug price itself but rather a subsidy provided by National Health Insurance, the health insurance finances inevitably worsen. This year alone, government subsidies to health insurance exceed 10 trillion KRW. Concerns are growing that continuous introduction of ultra-high-priced therapies could spark social controversy.

Both manufacturers argue that the high prices of these therapies are inevitable due to extensive technological development processes. Among them, Kymriah's price is further driven up by the characteristics of its manufacturing process. Kymriah is a cell therapy among the recently spotlighted gene and cell therapies (CGT). It was developed as the world's first CAR-T cell therapy.

Novartis' CAR-T Cell Therapy 'Kymriah'

The manufacturing process of Kymriah is complex. Cells from patients suffering from leukemia and other conditions are sent to Novartis' factory in the United States, where it takes about a month to produce the cell therapy. Genetic information is introduced into the patient's immune cells to enable them to recognize specific antigens on cancer cells and attack them. Unlike conventional anticancer drugs, the manufacturing method, efficacy, and administration differ entirely. It is a strictly personalized therapy made using the patient's own immune cells, known as an 'autologous' approach. Mass production is impossible, which inevitably drives up the price.

However, the effectiveness is clear. In studies involving patients with acute lymphoblastic leukemia, an impressive 82% of patients achieved complete remission (a state where no evidence of cancer is detected) within three months. Actual clinical statistics also show that more than half of the patients survive for over two years.

The problem arises when such personalized CGT therapies are introduced one after another. Because they are created by directly intervening in an individual's genes or cells, their efficacy is excellent, but mass production is impossible, leading to high prices. The limited number of patients who can actually receive treatment is why the global CAR-T cell therapy market remains at around 10 billion USD. Moreover, only six CAR-T cell therapies have been approved by the U.S. Food and Drug Administration (FDA), including Kymriah, Gilead's 'Yescarta' and 'Tecartus,' Bristol-Myers Squibb's (BMS) 'Breyanzi' and 'Abecma,' and Johnson & Johnson's 'Carvykti,' indicating a very small market.

Nevertheless, global pharmaceutical companies worldwide view CGT as the future growth engine and are fiercely competing in technology development. According to Evaluate Pharma, the global CGT market is expected to grow rapidly to 55.6 billion USD by 2026, with an average annual growth rate of 49.4%.

Industry experts believe that to overcome these challenges, efforts should continue to develop 'allogeneic' CGT therapies, which differ from autologous methods like Kymriah, to reduce prices and expand the market. Representative examples include Allogene's 'ALLO-715' for relapsed refractory multiple myeloma and 'UCART19' for acute lymphoblastic leukemia.

Big pharma companies are also actively developing allogeneic cell therapies. Roche has signed a large-scale licensing agreement with Adaptimmune to develop allogeneic T-cell receptor (TCR)-T cell therapies, and Gilead has partnered with Shoreline to develop allogeneic CAR-NK cell therapies. In South Korea, Vigencell is developing 'V-Ranger (VR)-CAR' using gamma delta T cells.

These allogeneic CAR-T therapies use immune cells cultured from blood stored in cell banks and can be administered to multiple patients, making them cheaper and allowing for rapid dosing. However, since they involve receiving blood from others, immune response management through immunosuppressants is necessary, which is a drawback.