Emergent treatments for β-thalassemia and orphan drug legislations

Enrico Costa; Maria Domenica Cappellini; Stefano Rivella; Adriana Chilin; Eva Alessi; Massimo Riccaboni; Hubert G.M. Leufkens; Lucio Luzzatto

doi:10.1016/j.drudis.2022.103342

Emergent treatments for β-thalassemia and orphan drug legislations

Enrico Costa^*, Maria Domenica Cappellini, Stefano Rivella, Adriana Chilin, Eva Alessi, Massimo Riccaboni, Hubert G.M. Leufkens, Lucio Luzzatto

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

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.

Original language	English
Article number	103342
Pages (from-to)	1-13
Number of pages	13
Journal	Drug Discovery Today
Volume	27
Issue number	11
DOIs	https://doi.org/10.1016/j.drudis.2022.103342
Publication status	Published - Nov 2022

Bibliographical note

Publisher Copyright:
© 2022 The Authors

Keywords

Beta thalassemia
Global health
Orphan drugs

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1-s2.0-S135964462200335X-mainFinal published version, 2.16 MBLicence: CC BY

Cite this

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title = "Emergent treatments for β-thalassemia and orphan drug legislations",

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.",

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AU - Costa, Enrico

AU - Cappellini, Maria Domenica

AU - Rivella, Stefano

AU - Chilin, Adriana

AU - Alessi, Eva

AU - Riccaboni, Massimo

AU - Leufkens, Hubert G.M.

AU - Luzzatto, Lucio

PY - 2022/11

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N2 - 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.

AB - 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.

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Emergent treatments for β-thalassemia and orphan drug legislations

Abstract

Bibliographical note

Keywords

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