オクヤマ　リョウ   OKUYAMA Ryo   奥山 亮    所属   立命館アジア太平洋大学  国際経営学部    職種   教授
言語種別	英語
発行・発表の年月	2023/04
形態種別	学術論文
査読	査読あり
標題	Modality diversification and best-in-class small-molecule drugs: Recent trend of orphan drug development.
執筆形態	単著
掲載誌名	Medicine in Drug Discovery
掲載区分	国外
出版社・発行元	Elsevier
巻・号・頁	18,pp.100156
総ページ数	6
担当区分	筆頭著者,責任著者
著者・共著者	Ryo Okuyama
概要	Pharmaceutical drug development has shifted to orphan diseases. This study investigated technology and development trend and strategy of orphan drugs approved by FDA from 2011 to 2022. Modality technology used for orphan drugs have been diversified, enhancing the drug development for orphan diseases that are otherwise challenging to treat with conventional drugs. Recently, orphan drugs using different modalities have been competitively developed with the same mechanism of action. In several cases, small-molecule drugs with unique characteristics offer advantages such as dosing convenience and non-genetic patient coverage over nucleic acid drugs, antibody drugs, and gene therapy.
DOI	https://doi.org/10.1016/j.medidd.2023.100156