Analysis of orphan designation status for FDA approved drugs, and case studies in oncology, neuroscience and metabolic diseases

[Display omitted] Many new drugs have been approved over the past decade for rare or orphan diseases. The passage of the Orphan Drug Act (ODA) in 1983 has provided key economic and regulatory incentives to provide medicines for patients who are suffering from rare diseases that may not be commercial...

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Bibliographic Details
Published inBioorganic & medicinal chemistry Vol. 80; p. 117170
Main Authors Brown, Dean G., Wobst, Heike J.
Format Journal Article
LanguageEnglish
Published England Elsevier Ltd 15.02.2023
Subjects
Approved drugs
Drug Approval
FDA
Humans
Metabolic Diseases
Orphan drug
Orphan Drug Act (ODA)
Orphan Drug Production
Rare disease
Rare Diseases - drug therapy
United States
United States Food and Drug Administration
United States
FDA
Orphan Drug Act (ODA)
Orphan drug
Rare disease
Approved drugs
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Summary:[Display omitted] Many new drugs have been approved over the past decade for rare or orphan diseases. The passage of the Orphan Drug Act (ODA) in 1983 has provided key economic and regulatory incentives to provide medicines for patients who are suffering from rare diseases that may not be commercially attractive for research and development. We have analyzed 497 novel drugs approved from 2010 - June 13, 2022, of which 220 were given orphan designation status. We discuss trends over this time period, potential risks for long development times, and provide example case studies of successful development and launch of novel drugs for rare diseases.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
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ObjectType-Review-1
ISSN:0968-0896
1464-3391
DOI:10.1016/j.bmc.2023.117170