IASO Bio Secures Significant Orphan Drug Designation for Novel CAR-T Therapy in South Korea

IASO Bio Achieves Orphan Drug Designation in South Korea for Equecabtagene Autoleucel



In a significant milestone for the biopharmaceutical company IASO Biotherapeutics, the South Korean Ministry of Food and Drug Safety has granted Orphan Drug Designation (ODD) to their innovative CAR-T cell therapy, Equecabtagene Autoleucel (Fucaso). This designation is aimed at treating adult patients suffering from relapsed or refractory multiple myeloma (R/R MM), particularly those who have undergone at least three previous therapies, including both a proteasome inhibitor and an immunomodulatory agent.

Understanding Orphan Drug Designation in South Korea


The dual criteria established by the Korean MFDS serves as a guideline for granting orphan drug status. Firstly, the drug must be intended for use in conditions affecting a domestic patient population of fewer than 20,000 individuals. Secondly, the therapy should either offer a significant improvement in safety or efficacy compared to existing treatments or address diseases for which no effective treatments are currently available. Furthermore, South Korea has provisions in place to facilitate the import of orphan drugs that are available internationally but not within the country.

The receipt of this designation marks an acceleration in the registration and approval process for Equecabtagene Autoleucel, holding promising implications for patients in Korea who are in desperate need of viable treatment options.

Quote from IASO Bio's Leadership


Jinhua Zhang, the Founder and CEO of IASO Biotherapeutics, expressed her optimism regarding this significant achievement, stating, “The Orphan Drug Designation for Equecabtagene Autoleucel in South Korea not only mirrors our global aspirations but also enhances our efforts to provide timely access to innovative cell therapies for patients. Following our earlier approval in Saudi Arabia, we are committed to executing our global registration strategy aimed at submitting applications across multiple countries in conjunction with each region's specific needs.”

The Therapeutic Need for Multiple Myeloma Treatments


Multiple myeloma (MM) is one of the most common blood cancers, ranked as the second-most prevalent hematological malignancy worldwide. According to Globocan data, the incidence rate in 2022 was approximately 1.8 cases per 100,000 people, with the five-year prevalence standing at 6.8 per 100,000 individuals. Current treatment options, while making strides, continue to fall short, with a high rate of relapse among patients and increased refractoriness to numerous drug classes. Thus, innovative therapies like Equecabtagene Autoleucel are critically needed to provide deeper responses and long-lasting remissions.

Insight into Equecabtagene Autoleucel (Fucaso)


Equecabtagene Autoleucel represents a state-of-the-art CAR-T cell therapy that utilizes lentivirus as a vector to modify autologous T cells. The therapy's design incorporates a fully human single-chain variable fragment (scFv), along with a CD8α hinge and transmembrane domain, 4-1BB co-stimulatory molecules, and CD3ζ activation domains. Following meticulous molecular screening and exhaustive in vitro and in vivo assessments, Equecabtagene Autoleucel has shown remarkable effectiveness paired with impressive long-term persistence, offering patients the possibility of sustained remission and protection against multiple myeloma.

About IASO Biotherapeutics


Founded to push the boundaries of cancer care, IASO Bio focuses on groundbreaking cell therapies and biologics targeting oncology and autoimmune diseases. The company is well-equipped at every stage of drug development — from early discovery to clinical validation, regulatory compliance, and market introduction. Currently, IASO Bio has a robust pipeline featuring over ten innovative treatments, including Equecabtagene Autoleucel, which acquired approval from China’s National Medical Products Administration in June 2023 and by the U.S. FDA’s IND in December 2022 for the treatment of R/R MM.

As IASO Bio strives to meet unmet medical needs in treatments, their ongoing commitment to innovation suggests a bright future for patients both locally in South Korea and on a global scale.

For further information, please visit IASO Bio or check their LinkedIn at IASO Biotherapeutics LinkedIn

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