Japan regulates the approval of regenerative medicine products and regenerative medicine treatments administered by medical institutions through separate laws and procedures. Regenerative medicine products must undergo PMDA review and obtain manufacturing and marketing approval from the Ministry of Health, Labor, and Welfare (MHLW) in accordance with the Pharmaceutical and Medical Device Act. The country also has a system whereby products with confirmed safety and reasonably predictable efficacy are granted conditional or time-limited approval, after which a decision on full approval is reassessed based on additional post-market data.
In contrast, the Act on the Safety of Regenerative Medicine (ASRM) regulates the procedures for medical institutions to provide cell therapy in clinical research or medical practice, as well as cell processing facilities and the operation of review committees. Medical institutions must prepare a plan for providing regenerative medicine prior to treatment, have it reviewed by the Committee on Recognized Regenerative Medicine, and submit it to the authorities. Cell processing can be performed within the medical institution or outsourced to an external cell processing facility that meets the required standards. Facilities located overseas may also process cells for use in regenerative medicine treatments conducted by Japanese medical institutions if they receive certification from the Ministry of Health, Labor and Welfare.
Japan’s CGT: Approval vs. Hospital-Based Regenerative MedicineOf course, Japan’s regulations are not lax. Medical institutions’ treatment plans, cell manufacturing processes, raw material management, transportation and storage, and quality and safety management systems are all subject to review. The higher the risk of a regenerative medicine treatment, the stricter the review and regulatory scrutiny become.
However, a key difference from South Korea is that even before product approval, medical institutions, cell processing facilities, and researchers can divide roles to design clinical and medical delivery models. From the perspective of South Korean companies, this means they can explore business opportunities to link local hospitals with their manufacturing and quality control capabilities, separate from long-term clinical trial and approval strategies for selling finished therapeutic products.
An official in the pharmaceutical and biotech industry stated, “Hospital-based regenerative medicine in Japan is not so much a pathway to drug approval as it is a separate pathway that allows for the joint design of cell manufacturing, quality control, and treatment operational systems with local partners under a treatment delivery plan,” adding, “Foreign companies can also turn this into a viable business if they establish local hospital networks and supply chains.”
Apart from hospital-based regenerative medicine, Japan has also been steadily expanding its market for commercialized products. Global CAR-T therapies such as Novartis’ “Kymriah,” Gilead’s “Yescarta,” Bristol-Myers Squibb’s “Breyanzi” and “Abecma,” and Janssen’s “Kavikti” have received approval in Japan. In March of this year, “Amchepri,” a treatment for Parkinson’s disease, and “Rehart,” a treatment for severe ischemic heart failure, received conditional and time-limited approval from Japan’s Ministry of Health, Labor and Welfare. These two products are the world’s first induced pluripotent stem cell (iPSC)-derived regenerative medicine products to receive approval for practical use.
South Korea Has Also Taken Its First Steps Toward Treatment, but Remains in the “Early Stages”Under the Advanced Regenerative Bio Act, South Korea distinguishes between advanced biopharmaceuticals and advanced regenerative medicine. To market products such as cell therapies, gene therapies, and tissue engineering products, manufacturers must obtain product approval from the Ministry of Food and Drug Safety (MFDS). The standard drug development pathway requires demonstrating safety and efficacy based on manufacturing processes, quality standards, and preclinical and clinical data. The Ministry of Food and Drug Safety (MFDS) also operates a conditional approval system for rare and severe diseases.
Advanced regenerative medicine is a separate system under which medical institutions conduct research or treat patients. The treatment system was introduced in February of last year, and in April of this year, a treatment plan for rare lymphoma submitted by Yeouido St. Mary’s Hospital of the Catholic University of Korea received the first approval of suitability. The plan involves administering ViGenCell Inc.’s autologous immune cell therapy “VT-EBV-N” to patients in complete remission who are at high risk of relapse, and actual patient treatment began last month.
However, in South Korea, advanced regenerative medicine treatments are still structured such that designated institutions carry out treatment plans that have been reviewed and approved for patients with serious, rare, or intractable diseases. Compared to countries like Japan, which have established business models by dividing roles between medical institutions and external cell processing facilities, the domestic market is still considered to be in its early stages.
MEDIPOST CO., LTD.(078160)“CartiStem” by [Company A] and “ImmunCell-LC” by GC CELL(144510)are leading domestically produced cell therapies that have accumulated prescription experience in Korea. However, since both products are non-covered therapies not eligible for national health insurance reimbursement, the financial burden on patients is significant. This, combined with the limited patient population eligible for treatment, acts as a constraint on market expansion. Cell therapies also incur higher production and quality control costs than conventional synthetic pharmaceuticals.
Another industry official stated, “Domestic cell therapies face significant challenges even after approval,” adding, “In a system centered on non-covered treatments, it is difficult to simultaneously increase patient access and production scale, and process innovations that can lower manufacturing costs are directly linked to competitiveness in overseas markets.”
The official continued, “In contrast, Japan features two coexisting pathways: one that targets the public insurance market through PMDA approval, and another that aligns manufacturing and supply capabilities with medical institutions’ regenerative medicine provision plans.” They noted, “From a corporate perspective, it is advantageous to be able to design entry strategies based on the maturity of their pipeline and their manufacturing capabilities.”
K-CGT: Beyond the Domestic Market, Heading to JapanJapan is recognized as a market of opportunity offering multiple entry routes for domestic companies. Strategies vary, including the finished therapeutic model—like MEDIPOST CO., LTD., which aims for product approval and insurance coverage through local clinical trials—the model—like Ipsell—that designs clinical trial and joint development structures in collaboration with hospitals and pharmaceutical companies, the model—like ENCell Co., Ltd. and GC CELL—that exports cell processing and quality control infrastructure, and the model—like Chabiotech Co.,Ltd.(085660)—that provides regenerative medicine services locally.
MEDIPOST CO., LTD. has chosen the most conventional path in Japan. Having completed the Phase 3 clinical trial in Japan for “CartiStem,” an allogeneic umbilical cord blood-derived mesenchymal stem cell therapy, the company is preparing to submit a marketing authorization application within the year. Based on domestic clinical data and in consultation with the PMDA, CartiStem skipped the Phase 1 and 2 clinical trials in Japan and proceeded directly to Phase 3. Since the company has announced that it met the key efficacy endpoints in the Japanese clinical trials, the focus of the upcoming review is expected to be on the interpretation of clinical data, manufacturing and quality data, and long-term safety management plans.
ENCell Co., Ltd. is reviewing its path to entering the Japanese market, weighing the options between clinical trials for marketing authorization and clinical research for regenerative medicine. Since 2024, ENCell has been discussing the possibility of conducting Japanese clinical trials for “Mucon,” an iPSC-derived osteoarthritis treatment, with Kamakura General Hospital near Shonan i-Park. While currently conducting a preliminary analysis to prepare for PMDA review, the company is evaluating whether to proceed with clinical trials for marketing authorization or to first conduct clinical research under regenerative medicine laws.
ENCell Co., Ltd. and GC CELL have adopted a strategy of first leveraging their cell processing and quality control capabilities in the Japanese market, rather than pursuing drug marketing authorization. Last May, ENCell Co., Ltd. received certification from Japan’s Ministry of Health, Labor and Welfare (MHLW) for its Hanam GMP Plant No. 2 as a manufacturing facility for specific cell processing products. The company is promoting a model in which Japanese medical institutions handle patient care and submit applications for regenerative medicine treatment plans, while ENCell Co., Ltd. is responsible for cell manufacturing, supply, quality control, and related documentation support.
GC CELL also received certification as a manufacturing facility for specific cell processing products from Japan’s Ministry of Health, Labor and Welfare that same month. Following an on-site inspection by the PMDA late last year, the company was recognized for having manufacturing facilities and a quality management system that comply with Japanese standards. GC CELL plans to use this certification as a foundation for the Japanese market entry of its commercially available domestic products—ImmunCell LC and the CD5-targeted CAR-NK therapy “GCC2005”—as well as for expanding its local CDMO business.
Chabiotech Co.,Ltd. is providing regenerative medicine services using autologous adipose-derived stem cells and autologous NK cells (immune cells) in collaboration with “Total Cell Clinic Tokyo (TCC),” a regenerative medicine hospital it operates in Japan. Total Cell Clinic Tokyo has provided more than 10,000 regenerative medicine treatments to date using cultivation technology patented in Japan at a facility that, in 2015, became the first foreign-owned company to obtain approval from the Ministry of Health, Labor and Welfare for regenerative medicine-related services.
