Emergent treatments for β-thalassemia and orphan drug legislations
Publication date
2022-11
Authors
Costa, Enrico
Cappellini, Maria Domenica
Rivella, Stefano
Chilin, Adriana
Alessi, Eva
Riccaboni, Massimo
Leufkens, H.G.
Luzzatto, Lucio
Editors
Advisors
Supervisors
Document Type
Article
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cc_by
Abstract
In many countries, β-thalassemia (β-THAL) is not uncommon; however, it qualifies as a rare disease in the US and in European Union (EU), where thalassemia drugs are eligible for Orphan Drug Designation (ODD). In this paper, we evaluate all 28 ODDs for β-THAL granted since 2001 in the US and the EU: of these, ten have since been discontinued, twelve are pending, and six have become licensed drugs available for clinical use. The prime mover for these advances has been the increasing depth of understanding of the pathophysiology of β-THAL; at the same time, and even though only one-fifth of β-THAL ODDs have become licensed drugs, the ODD legislation has clearly contributed substantially to the development of improved treatments for β-THAL.
Keywords
Beta thalassemia, Global health, Orphan drugs, Pharmacology, Drug Discovery
Citation
Costa, E, Cappellini, M D, Rivella, S, Chilin, A, Alessi, E, Riccaboni, M, Leufkens, H G M & Luzzatto, L 2022, 'Emergent treatments for β-thalassemia and orphan drug legislations', Drug Discovery Today, vol. 27, no. 11, 103342, pp. 1-13. https://doi.org/10.1016/j.drudis.2022.103342