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

Shaohong WANG; Wei ZUO; Xin LIU; Limei LI; Bo ZHANG

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

VernacularTitle:2024年度美国、欧盟、日本上市孤儿药概览与分析
Author: Shaohong WANG ¹ ; Wei ZUO ¹ ; Xin LIU ¹ ; Limei LI ² ; Bo ZHANG ³
Author Information

1. Department of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;.
2. Clinical Pharmacology Research Center, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;.
3. Department of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;National Center for Medical Quality Control of Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China;.
Publication Type:Journal Article
Keywords: rare diseases; orphan drugs; the United States; the European Union; Japan
From: JOURNAL OF RARE DISEASES 2025;4(4):472-477
CountryChina
Language:Chinese
Abstract:
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.