On July 9, 2025 IASO Biotherapeutics ("IASO Bio"), a biopharmaceutical company focused on the discovery, development, manufacturing, and commercialization of innovative cell therapies and biologics, reported that its self-developed anti-BCMA CAR-T cell therapy product, Equecabtagene Autoleucel (Fucaso), has been granted Orphan Drug Designation (ODD) by the Ministry of Food and Drug Safety (MFDS) of South Korea for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have received at least three prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent (Press release, IASO Biotherapeutics, JUL 9, 2025, View Source [SID1234654316]).
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Currently, the Korean MFDS adopts a dual standard for orphan drug designation: 1. Drugs used for diseases with a domestic patient population (prevalence) of 20,000 or fewer; 2. Drugs used for diseases for which appropriate treatment methods or drugs have not been developed, or drugs that demonstrate significantly improved safety or efficacy compared to existing alternative drugs. For orphan drugs that are "available abroad but not domestically", South Korea has policies to facilitate their import*. Following ODD, the registration and approval process of Equecabtagene Autoleucel Injection in South Korea is expected to be accelerated, potentially enabling earlier access for local patients.
Ms. Jinhua Zhang, Founder, Chairwoman, and CEO of IASO Biotherapeutics, stated:
"The Orphan Drug Designation granted to Equecabtagene Autoleucel Injection in South Korea marks another significant international regulatory milestone for the product, following its ODD approval in Saudi Arabia in May. This Designation is expected to accelerate patient access to this CAR-T therapy in South Korea. IASO Bio is implementing its global registration strategy of ‘parallel multi-country submissions with regional synergy’to obtain approvals for this innovative cell therapy in additional countries and regions, ultimately extending its benefits to a wider patient population."
About Multiple Myeloma（MM)
Multiple myeloma (MM) is the second most common hematological malignancy globally. According to Globocan data, the global incidence of multiple myeloma in 2022 was 1.8 per 100,000 people, with a 5-year prevalence of 6.8 per 100,000. Despite progress in current anti-myeloma treatments, MM remains largely incurable with multiple relapses and tendency to develop refractoriness to several drug classes, presenting a major therapeutic challenge. Thus, there is an unmet need for new treatment options beyond these current anti-myeloma therapies for the treatment of relapsed or refractory MM, capable of achieving deep and durable responses.
About Equecabtagene Autoleucel（Fucaso）
Equecabtagene Autoleucel（Fucaso） is an innovative fully human anti-BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully human scFv, CD8a hinge and transmembrane domain, and 4-1BB co-stimulatory molecule and CD3ζactivation domains. Based on rigorous molecular structure screening and comprehensive in vitro and in vivo functional evaluations, Fucaso demonstrates rapid and potent efficacy, accompanied by exceptional long-term persistence in vivo, enabling patients to achieve deep and durable remission,providing continuous protection and care for patients with multiple myeloma.