Department   Ritsumeikan Asia Pacific University  College of International Management
   Position   Professor
Language English
Publication Date 2023/04
Type Research paper
Peer Review Peer reviewed
Title Modality diversification and best-in-class small-molecule drugs: Recent trend of orphan drug development.
Contribution Type Single Work
Journal Medicine in Drug Discovery
Journal TypeAnother Country
Publisher Elsevier
Volume, Issue, Page 18,pp.100156
Total page number 6
Authorship Lead author,Corresponding author
Author and coauthor Ryo Okuyama
Details 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