Korea's FDA strengthens biopharmaceutical approval for enhanced clinical support

The top-selling immuno-oncology drug 'Keytruda', which has held the number one sales position for 10 years since its launch, the rheumatoid arthritis treatment 'Humira', and the recently explosive-demand weight loss drug 'Wegovy' are all biopharmaceuticals made from substances derived from humans or other organisms. They are gaining more market share recently due to fewer side effects and better therapeutic effects compared to chemically synthesized traditional medicines.

According to the '2024 Biopharmaceutical Industry Trends Report' from the Korea Bio Association, the global biopharmaceutical market is projected to grow at an annual rate of 9% from 663 trillion won in 2023 to 1,114 trillion won by 2029. The domestic biopharmaceutical market is also growing, with production results of gene recombinant medicines, including biosimilars, entering the 2 trillion won range for the first time in history. Biopharmaceuticals include gene recombinant medicines, cell and gene therapies (CGT), cell culture medicines, and vaccines.

The government has also stepped up to promote biopharmaceuticals. The Korea Pharmaceutical and Biologics Agency under the Ministry of Food and Drug Safety held a '2025 Biopharmaceutical Licensing and Review Capacity Building Seminar' on the 17th at the ST Center in Gangnam, Seoul, targeting industry stakeholders, and announced this year's work plan, including the establishment of guidelines.

Choi Young-joo, head of the Biologic Review Department, noted, "We have begun discussions for World Health Organization (WHO) designation as a regulatory authority for pharmaceutical excellence and for joint evaluations with the European Medicines Agency (EMA)," and added, "We will actively support domestic pharmaceutical companies and research institutions to facilitate clinical entry and ensure licenses for domestically produced biopharmaceuticals."

Initially, for gene recombinant medicines, a standardization plan will be put in place for review criteria that meet global standards to support the development and approval of biosimilars. Guidance on information presentation formats related to proving equivalence with original medicines will be provided by November. The agency is also working with the U.S. Food and Drug Administration (FDA) to create new international guidelines for simplifying clinical evaluations of biosimilars through the International Council for Harmonisation (ICH).

The review process for antibody-drug conjugates (ADC), which are actively being developed worldwide, will also be reformed. Guidelines for quality evaluation of ADC candidate substances are set to be established in November. ADCs are innovative targeted therapies known as guided missile cancer drugs that deliver drugs only to cancer cells.

The agency will also revise regulatory guidelines in the CGT field for CAR-T (Chimeric Antigen Receptor T-cell) therapies, gene editing technologies (CRISPR-Cas9), and stem cell therapies. In June of this year, guidelines for immune toxicity evaluation of allogeneic stem cell therapies will be created, and in October, the considerations for evaluating dendritic cell therapies for cancer treatment and guidelines for quality control testing of cell therapies will be released.

For vaccines, guidelines for evaluating combinations of diphtheria, tetanus, and pertussis vaccines (Tdap) will be revised in June. The plan includes reflecting detailed content, including consistency verification in the manufacturing and testing method validation to apply a single dilution method. Although the Tdap vaccine is classified as a national essential vaccination, the vaccines distributed in the country rely entirely on imports. Currently, GC Biopharma is conducting phases 1 and 2.

Last month, a public-private consultative body was formed for joint reviews (OPEN), and quarterly meetings will be held starting this month. A vaccine clinical support consultative body is also planned to operate within the year. For items entering clinical trials among companies developing the country's first vaccine platform, technical support will be provided, including step-by-step clinical design.