South Korean biotechnology company MediPost has entered full-scale Phase 3 clinical testing in the United States for its landmark knee osteoarthritis treatment, Cartistem. The company announced on July 8 that it has successfully completed the first patient enrollment, treatment, and dosing for the U.S. Phase 3 clinical trial of Cartistem, its allogeneic mesenchymal stem cell therapy derived from human umbilical cord blood.
The multi-center trial is set to enroll 300 patients diagnosed with knee osteoarthritis and will be conducted across approximately 70 clinical trial sites throughout the United States and Canada. Patient enrollment and clinical dosing will take place over a two-year window, followed by an additional two-year post-treatment follow-up period to comprehensively evaluate both long-term joint structure improvements and the relief of clinical symptoms. Upon successful completion of the trial, MediPost plans to compile a comprehensive clinical study report (CSR) and submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA).
With this clinical push, MediPost aims to definitively confirm whether Cartistem can halt or reverse the progression of knee osteoarthritis through direct cartilage regeneration. The company is positioning the treatment as a disease-modifying osteoarthritis drug (DMOAD)—a highly coveted class of therapeutics that goes beyond palliative pain management to actively repair cartilage and restore joint structures, thereby suppressing the root cause of disease progression.
Leveraging Real-World Evidence for a Streamlined FDA Pathway
Notably, this U.S. Phase 3 trial is moving forward under a rare and highly advantageous regulatory framework, having received FDA clearance to proceed as a "single pivotal study."
Typically, the FDA requires a minimum of two independent, large-scale Phase 3 clinical trials before evaluating a novel drug for regulatory approval. However, MediPost secured an exception for a single trial by presenting a robust package of existing clinical data accumulated from successful late-stage trials in South Korea and Japan. This was bolstered by long-term real-world evidence (RWE) extracted from treatment tracking data on approximately 560 South Korean patients who have been monitored for more than three years post-dosing. Industry analysts note that the company also benefited from designing a rigorous, FDA-compliant "adequate and well-controlled clinical trial" protocol that met strict regulatory standards.
Cartistem originally received regulatory approval from South Korea's Ministry of Food and Drug Safety in January 2012, with the first commercial procedures launching in April of that year. Now in its 14th year of commercial availability, the therapy has established a dominant presence in South Korea as a premier stem cell option for severe knee osteoarthritis. Meanwhile, in Japan, Cartistem has successfully completed its localized Phase 3 trials, and the company is actively preparing to file for official Japanese marketing authorization by the end of 2026.
“Commencing patient enrollment and initial dosing in this U.S. Phase 3 trial represents a critical milestone for our entry into the global pharmaceutical market,” said Lee Seung-jin, Head of MediPost's Global Business Division. “At the same time, leading groups of prominent orthopedic specialists across the United States are demonstrating exceptionally strong interest in the cartilage regeneration and tissue remodeling outcomes achieved by Cartistem.”
