Iaso Bio's Fucaso (Equecabtagene Autoleucel) Receives Orphan Drug Designation in South Korea for Relapsed/Refractory Multiple Myeloma

Jul 09, 2025 18:00 CST Updated 18:00

IASO Biotechnology

Cancer Treatment New Drug Developer

On July 9, 2025, IASO Bio announcedSouth Korea's Ministry of Food and Drug Safety (MFDS) has granted Orphan Drug Designation (ODD) to IASO Bio's self-developed BCMA-targeted CAR-T cell therapy product, Fucaso Injection (Fucaso)., used to treat adult patients with relapsed or refractory multiple myeloma (R/R MM) who have experienced disease progression after at least three prior lines of therapy (including at least one proteasome inhibitor and one immunomodulatory agent).

Currently, the MFDS in South Korea defines orphan drugs according to the following criteria: 1) The number of patients affected is less than 20,000; 2) The drug is used to treat diseases for which no appropriate therapy has been developed, or its safety/effectiveness is significantly higher compared to existing alternative therapies. For orphan drugs that are available overseas but not yet in China, South Korea encourages the importation of such orphan drugs.^*。Iaso Bio's Ixazomib Injection Expected to Accelerate Registration and Approval in Korea After Receiving ODD, Benefiting Local Patients Soon.

Ms. Zhang Jinhua

Founder, Chairman and Chief Executive Officer of IASO Bio

This orphan drug designation for IASO Bio's Iciclenogene Icreleucel Injection in South Korea marks another significant international regulatory recognition for the product, following its ODD approval in Saudi Arabia this past May. This designation is expected to accelerate the accessibility of this CAR-T therapy in South Korea.IASO Bio is advancing the international registration strategy of 'multi-country parallel, regional synergy' to promote the approval and market launch of this innovative cell therapy in more countries and regions., benefiting a wider patient population.

* "Research on the Supply Guarantee System of Orphan Drugs in South Korea from the Perspective of Availability and Its Implications" by Yang Yifan, Xie Jinping, Weng Tingting, Yan Liyu, Shao Rong (National Drug Policy and Pharmaceutical Industry Economic Research Center, China Pharmaceutical University, Nanjing 211198)

About Multiple Myeloma

Multiple Myeloma (MM) is the second most common hematologic malignancy globally. According to Globocan data, the global incidence rate of multiple myeloma was 1.8/100,000 in 2022, with a 5-year prevalence rate of 6.8/100,000. Despite advances in current anti-myeloma treatments, MM remains largely incurable, prone to multiple relapses, and tends to develop resistance to various drug classes, presenting significant challenges for treatment. Therefore, for the treatment of relapsed or refractory MM, there is still an urgent need for new therapeutic options beyond existing anti-myeloma therapies to achieve deep and durable responses.

About Icaros Injection (Fucaso)

Fucaso (Equecabtagene Autoleucel Injection) is a CAR-T cell therapy targeting B-cell maturation antigen (BCMA). It uses lentivirus as a gene vector to transfect autologous T cells. The CAR comprises a fully human scFv, CD8a hinge and transmembrane domain, 4-1BB co-stimulatory molecule, and CD3ζ activation domain. Based on rigorous molecular structure screening and comprehensive in vivo and in vitro functional evaluation, Fucaso...^®With rapid and potent efficacy, as well as remarkable persistence in the body, it enables patients to achieve deep and sustained remission, continuously protecting patients with multiple myeloma.

About IASO Bio

IASO Bio, established in 2017, is a leading biopharmaceutical company focusing on the research, production, and sales of innovative cell therapies. The company takes the development of hematological tumor cell drugs as its innovative cornerstone and expands into the fields of autoimmune diseases and antibody drugs, possessing comprehensive capabilities covering the entire process from early drug discovery, clinical development, regulatory submission to commercialization. IASO Bio currently has more than 10 innovative drug candidates at different stages of development, including the world's first fully human CAR-T product targeting BCMA, the Idecabtagene Vicleucel Injection (Focuxin), which was independently developed by IASO Bio.^®) was approved for marketing by the China National Medical Products Administration (NMPA) in June 2023, for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have progressed after at least three prior lines of therapy (including at least one proteasome inhibitor and one immunomodulatory agent). Its frontline indication for treating 2-3 line multiple myeloma (MM) has entered Phase III clinical trials. In addition, Ixazomib Injection has received multiple Investigational New Drug (IND) approvals in both China and the United States for autoimmune diseases.

At the same time, CT120, a dual-target (CD19/CD22) product for lymphoma, is about to enter Phase II clinical trials. Another product targeting GPRC5D for the treatment of relapsed or refractory multiple myeloma, IASO118, has received IND approval in China. In addition to cell therapy products, the company's fully human anti-CD19 antibody product, IASO-782, has received IND approval in both China and the United States for the treatment of multiple autoimmune diseases, while still exploring more indications for autoimmune diseases. The company has successively partnered with overseas cell therapy company Sana.Therapeutics, Cabaletta Bio, and Umoja Biopharma have reached global business development licensing or R&D collaborations, actively exploring the development of next-generation cell therapy products. Relying on its strong management team, innovative product pipeline, in-house GMP manufacturing, and advanced clinical development capabilities, IASO Bio is committed to bringing transformative, accessible innovative therapies and the hope of a cure to patients in China and around the world. For more information, please visit the company's official website: www.iasobio.com or LinkedIn page: www.linkedin.com/company/iasobiotherapeutics.