Department Ritsumeikan Asia Pacific University College of International Management Position Professor
|Modality diversification and best-in-class small-molecule drugs: Recent trend of orphan drug development.
|Medicine in Drug Discovery
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|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.