Overview and Analysis of Orphan Drugs Approved for Marketing in the United States, the European Union, and Japan in 2024

This paper conducts a pooled analysis of information related to orphan drugs approved for marketing in 2024 by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Pharmaceuticals and Medical Devices Agency (PMDA) of Japan. It systematically sorts out the scope of indications, therapeutic area distribution, and special review and approval pathways of these drugs, as well as their research and development progress and marketing status in China, thus providing a reference for researchers, regulatory authorities, and industry stakeholders in the field of rare disease drugs.