IASO Biotherapeutics (IASO Bio), a biopharmaceutical company focused on the discovery, development, manufacturing, and commercialization of innovative cell therapies and biologics, announced that its self-developed BCMA-targeted CAR-T cell therapy product, Equecabtagene Autoleucel (FUCASO), has been granted Orphan Drug Designation (ODD) by the Saudi Food and Drug Authority (SFDA) 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.
The ODD is a special regulatory incentive established by the Saudi SFDA to encourage the development of therapies for rare diseases. Drugs receiving this designation are eligible for expedited regulatory review and assistance with product development. With the ODD approval, Equecabtagene Autoleucel can now directly submit a New Drug Application (NDA) in Saudi Arabia, which is expected to shorten the approval process and enable earlier access for patients in the Middle East region.
Jinhua Zhang, founder, chairperson, and CEO of IASO Biotherapeutics, stated: “We are delighted that Equecabtagene Autoleucel has been grated the Orphan Drug Designation by the SFDA. This once again demonstrates international regulatory recognition of the clinical value of this innovative therapy. Saudi Arabia serves not only as a crucial starting point for our extension into the Middle East market, but also represents a significant step in our steady strategy of “Go Global”. We will work closely with local regulatory authorities, medical institutions and partners to advance the approval processes of Equecabtagene Autoleucel in Saudi Arabia, with the goal of delivering clinical benefits to patients in these regions.”
Multiple myeloma (MM) is the second most common haematological 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.
Equecabtagene Autoleucel 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 CD3?activation 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.
Founded in 2017, IASO Bio is a leading biopharmaceutical company specializing in the discovery, development, manufacturing, and commercialization of cutting-edge cell therapies and biologics. Initially focused on treatments for blood cancers, the company has strategically expanded into autoimmune diseases.