Administrative Action Challenging Rejection of Marketing Authorization for Stem Cell Therapy

Lee & Ko is representing R Bio Co., Ltd. (“R Bio”) in an administrative lawsuit seeking to revoke the decision of the Ministry of Food and Drug Safety (“MFDS”) rejecting R Bio’s application for marketing authorization (“MA”) for its stem cell therapy, JointStem. R Bio has developed JointStem over a period of approximately sixteen years since 2005.

This case squarely challenges the legality of the MFDS’s refusal to grant MA for a stem cell therapy whose clinical efficacy has been objectively demonstrated. The dispute raises fundamental questions concerning the rationality, transparency, and predictability of regulatory standards applicable to regenerative medicine products. It also carries significant implications for the broader regulatory environment governing Korea’s biotechnology and pharmaceutical industries.

In this matter, Lee & Ko is drawing upon its expertise in clinical biostatistics and pharmaceutical regulatory standards to develop a litigation strategy grounded in scientific rigor. The firm’s analysis addresses complex scientific and statistical issues, including: the appropriateness of the clinical trial design (Study Design), development and implementation of the Statistical Analysis Plan (SAP), hypothesis testing for primary and secondary endpoints, interpretation of efficacy and safety indicators, and the distinction between statistical significance and clinical relevance. In particular, with respect to the MFDS’s assessment of clinical efficacy, Lee & Ko’s arguments extend beyond a narrow inquiry into whether certain numerical endpoints achieved statistical significance. The firm is comprehensively examining the validity of the overall study design, the appropriateness of the patient population, the methodological soundness of the statistical testing framework, and the interpretability of long-term follow-up data. Through this approach, the case fundamentally contests whether the regulatory determination is consistent with accepted scientific and statistical principles.

More than a conventional administrative challenge to a marketing authorization rejection, this matter represents a pivotal examination of Korea’s regulatory standards for stem cell therapies and regenerative medicine products, including their alignment with international practice. The outcome is expected to provide an important reference point for future approval and regulatory processes concerning innovative drugs and advanced biopharmaceuticals.

Through its representation in this case, Lee & Ko further demonstrates its capabilities in bio/healthcare IP and regulatory disputes, including data-driven advocacy, sophisticated clinical data analysis, and strategic litigation at the intersection of science, regulation, and administrative law.