JointStem: A Road Closed by Korea’s MFDS, Now Opening in the United States
By Lee Sang-ki
AsiaN reporter, former president of the Korea Journalists Association
SEOUL: Nature Cell, a leading Korean biotech company, is accelerating its entry into the U.S. market with its stem cell therapy, JointStem. On the surface, this is a global expansion strategy, but beneath it lies a stark reality: the high and opaque clinical standards imposed by Korea’s Ministry of Food and Drug Safety (MFDS). After repeated setbacks at home, the company has turned abroad—a move that reveals the structural challenges of Korea’s regulatory environment.
Repeated Setbacks with Korea’s MFDS
In 2018, Nature Cell applied for conditional approval, and again in 2021 for full approval of JointStem. Both applications were rejected by the MFDS. Even after expanding the number of trial participants and submitting five-year follow-up data, the agency concluded that while the therapy showed statistical significance, its “clinical meaningfulness” was insufficient. The problem is that this criterion appeared unpredictable and uniquely applied in Korea without clear guidelines. In response, Nature Cell filed an administrative lawsuit and a claim for damages, directly challenging what it viewed as an unreasonable judgment by the MFDS.
A New Path in the United States
The U.S. Food and Drug Administration (FDA), by contrast, adopted a different approach. JointStem has been designated as both a Breakthrough Therapy (BTD) and a Regenerative Medicine Advanced Therapy (RMAT), giving it access to expedited review and more flexible procedures. This means that approval may proceed if medical need, safety, and the absence of alternatives are recognized, even if clinical outcomes are not perfect. Nature Cell plans to hold an End of Phase 2 meeting with the FDA in November, begin Phase 3 trials early next year, and pursue accelerated approval.
Building the U.S. Roadmap
Nature Cell is also pursuing regenerative medicine projects in states like Florida and Texas, which allow stem cell therapies outside federal approval, in order to secure early revenue. In addition, the company has announced plans to establish a research and production hub in Baltimore, Maryland, with an investment of \$300 million by 2031. This facility is expected to produce treatments for one million patients annually, with projected revenues of \$10 billion. “We will move beyond the barriers of Korea’s MFDS and establish ourselves as a global standard in the world’s largest market,” Chairman Ra Jeong-chan declared.
Challenges and Policy Implications
Nature Cell’s U.S. strategy is both an opportunity and a paradox. A therapy rejected by Korea’s MFDS for “insufficient effect” is now moving forward in the United States. To be sure, the MFDS’s strict standards reflect a commitment to patient safety and credibility. Yet when regulations become overly rigid and unpredictable, they risk stifling innovation. Is it right that companies are driven abroad to pursue breakthroughs they could not achieve at home?
This case poses serious questions about how Korea’s MFDS interprets and enforces its mandate. How should “patient-perceived effectiveness” be defined? How can Korea align with global regulatory standards while ensuring safety? Whether to close the door on innovation or to open it toward both patients and industry—the choice now rests with Korea’s MFDS.
