<Seoul Administrative Court Revokes MFDS Rejection of Marketing Authorization Application for Stem Cell Therapy>
1. Lee & Ko Successfully Represents Pharmaceutical Company in Challenge to MFDS’s Rejection of Cell Therapy Marketing Authorization Application
Lee & Ko successfully represented a pharmaceutical company in an administrative lawsuit challenging the Ministry of Food and Drug Safety (MFDS)’s rejection of the marketing authorization (MA) application for the stem cell therapy Jointstem, thereby paving the way for the osteoarthritis stem cell therapy—whose application had twice been rejected—to undergo a renewed review for MA
The Seoul Administrative Court ruled in favor of RNL Regenerative Medical Institute. (formerly Albio Co., Ltd., hereinafter referred to as “RNL Research Institute”) in its lawsuit seeking to revoke the MFDS’s rejection of its MA application for the stem cell therapy.
After completing its Phase III clinical trial under an MFDS-approved protocol, RNL Research Institute applied for marketing authorization (MA) in 2021. The MFDS rejected the application in 2023 for lack of "clinical significance" and, despite supplemental data submitted in 2024, rejected the renewed application again in 2025 on the same ground.
The MFDS based its decision on the view that the product lacked clinical significance because it failed to demonstrate superior therapeutic efficacy over existing therapies.
2. The Seoul Administrative Court Holds That the Pharmaceutical Affairs Act Requires Only Safety and Efficacy, Not Superiority Over Existing Therapies
The Court held that the central issue was not a highly technical or scientific determination entitled to judicial deference, but rather a legal question concerning the proper interpretation of “clinical significance," which underpins the assessment of a pharmaceutical’s safety and efficacy. It further held that where statistical significance has been established pursuant to a pre-approved clinical trial protocol, the relevant regulations should be interpreted as recognizing the product's safety and efficacy.
According to the court, requiring proof of superior efficacy over existing therapies as a condition for marketing authorization would effectively protect incumbent products and restrict the market entry of new pharmaceuticals. Because such a requirement would limit free competition and the freedom to conduct business, it must have an explicit statutory basis under the constitutional principle that restrictions on fundamental rights must be prescribed by law.
The Court emphasized that the Pharmaceutical Affairs Act (PAA) requires only that a pharmaceutical demonstrate safety and efficacy as conditions for marketing authorization and does not impose an additional requirement that the product be superior to existing therapies. It further concluded that interpreting “clinical significance” to require proof of superiority constitutes an arbitrary interpretation inconsistent with both the purpose of the PAA and the constitutional principle requiring a statutory basis for restrictions on fundamental rights.
The Court also found that, despite the fact that the Phase III clinical trial had already demonstrated statistical significance, the MFDS and the Central Pharmaceutical Affairs Council improperly denied MA based on opinions that were subject to concerns regarding potential conflicts of interest among certain committee members.
3. Lee & Ko’s Pharmaceutical and Biotechnology Litigation Expertise Secures Renewed Review of Domestic Cell Therapy
Drawing on its extensive experience in pharmaceutical regulatory and biotechnology litigation, Lee & Ko successfully argued that it was unlawful for the MFDS to deny the product’s clinical significance based on arbitrary criteria lacking any legal basis or pre-established regulatory standard, despite the product having demonstrated scientific and statistical validity. As a result, the firm secured a judgment from the Seoul Administrative Court holding that the rejection of the MA application was unlawful.
The decision underscores that, even in areas involving scientific and technical expertise where administrative agencies are afforded broad discretion, arbitrary interpretations that depart from the governing legal framework and restrict fundamental rights are impermissible. The ruling is expected to enhance legal predictability in pharmaceutical marketing authorization reviews, safeguard the freedom to conduct business, and broaden treatment options for patients.
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Keum Nang PARK ([email protected])
Donghyuk KANG ([email protected])
Eunkyoung LYU ([email protected])
Soo Yeon PARK ([email protected])
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