Saudi FDA Grants Orphan Drug Status to IASO Bio's Equecabtagene Autoleucel

SHANGHAI, NANJING, China and SAN FRANCISCO, May 9, 2025 /PRNewswire/ -- IASO Biotherapeutics, a biopharmaceutical firm dedicated to the advancement of innovative cell therapies and biologics, has secured Orphan Drug Designation (ODD) from the Saudi Food and Drug Authority (SFDA) for its proprietary BCMA-targeted CAR-T cell therapy, Equecabtagene Autoleucel (FUCASO). This therapy is aimed at adult patients with relapsed or refractory multiple myeloma (R/R MM) who have undergone at least three prior treatment lines, incorporating a proteasome inhibitor and an immunomodulatory agent.

The Saudi SFDA's ODD is a strategic regulatory incentive to spur the development of treatments for rare diseases. Therapies granted this status benefit from accelerated regulatory reviews and guidance during product development. With the ODD approval, Equecabtagene Autoleucel is now eligible for a direct New Drug Application (NDA) submission in Saudi Arabia, potentially expediting the approval timeline and facilitating quicker patient access in the Middle East.

Ms. Jinhua Zhang, Founder, Chairperson, and CEO of IASO Biotherapeutics, expressed her satisfaction with the designation, saying it underscores the international acknowledgment of the clinical significance of this cutting-edge therapy. Saudi Arabia is not only a pivotal entry point into the Middle Eastern market but also a crucial phase in IASO Bio's strategic international expansion. The company is committed to collaborating with local regulatory bodies, healthcare institutions, and partners to expedite the approval of Equecabtagene Autoleucel in Saudi Arabia, with the ultimate goal of bringing clinical advantages to patients in these areas.

Multiple myeloma (MM) ranks as the second most prevalent blood cancer globally. According to data from Globocan, the incidence rate of multiple myeloma in 2022 was 1.8 per 100,000 people worldwide, with a 5-year prevalence of 6.8 per 100,000. Although there have been advancements in existing treatments for this cancer, MM remains mostly incurable, often resulting in relapses and resistance to various drug classes, thus posing a significant therapeutic challenge. Consequently, there is a pressing need for new therapeutic options beyond current anti-myeloma treatments to address relapsed or refractory MM, aiming for deep and long-lasting responses.

Equecabtagene Autoleucel represents a groundbreaking fully human anti-BCMA CAR-T cell therapy that uses lentivirus as a gene delivery vector to modify patients' autologous T cells. The therapy incorporates a fully human single-chain variable fragment (scFv), a CD8a hinge and transmembrane domain, as well as 4-1BB co-stimulatory and CD3ζ activation domains. Through meticulous molecular structure selection and comprehensive functional evaluations both in vitro and in vivo, FUCASO has shown rapid and robust efficacy, along with outstanding long-term presence in the body, helping patients achieve deep and lasting remission. This offers continuous protection and care for individuals with multiple myeloma.

IASO Biotherapeutics focuses on discovering and developing innovative cell therapies and biologics for cancer and autoimmune conditions. The company encompasses a wide range of capabilities, covering every stage of drug development, from initial discovery to clinical trials, regulatory approval, and commercialization. Its pipeline includes over 10 novel products, among them Equecabtagene Autoleucel. This product received Biologics License Application (BLA) approval from China's National Medical Products Administration in June 2023 and U.S. FDA IND approval for treating R/RMM in December 2022. With a strong management team, innovative product lineup, and integrated manufacturing and clinical capabilities, IASO Biotherapeutics aims to provide transformative and cost-effective treatments that address unmet medical needs for patients in China and globally.