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Orphan Drugs and Their Impact on Pharmaceutical Development
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Functional Pharmacology.
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Immunology, Genetics and Pathology, Medicinsk genetik och genomik.
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Neuroscience, Functional Pharmacology.ORCID iD: 0000-0001-7112-0921
2018 (English)In: TIPS - Trends in Pharmacological Sciences, ISSN 0165-6147, E-ISSN 1873-3735, Vol. 39, no 6, p. 525-535Article, review/survey (Refereed) Published
Abstract [en]

High levels of productivity, with an increasing number of approvals for new molecular entities (NMEs) by the FDA during the past decade, have coincided with the emergence of innovative drugs for treatments of rare diseases that have utilized the FDA orphan drug program. Since 2000, NMEs with orphan designation encompass a significant portion of approved drugs and constitute about 80% of the approved drugs that have established novel human genome-encoded products in recent years. Biological approvals are also expanding, with 40% of the approved biological agents having orphan designation. This trend illustrates a pivot within the pharmaceutical industry: from research programs that focus on canonical blockbuster indications and targets, towards the establishment of new treatments for rare and difficult to treat diseases.

Place, publisher, year, edition, pages
2018. Vol. 39, no 6, p. 525-535
National Category
Pharmacology and Toxicology
Identifiers
URN: urn:nbn:se:uu:diva-358124DOI: 10.1016/j.tips.2018.03.003ISI: 000432349900001PubMedID: 29779531OAI: oai:DiVA.org:uu-358124DiVA, id: diva2:1241806
Available from: 2018-08-24 Created: 2018-08-24 Last updated: 2019-03-15Bibliographically approved

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