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, Enrico Costa, Maria Domenica Cappellini, Stefano Rivella, Adriana Chilin, Eva Alessi, Massimo Riccaboni, Hubert G M Leufkens, Lucio Luzzatto
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.
Conflict of interest statement
Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
Copyright © 2022 The Authors. Published by Elsevier Ltd.. All rights reserved.
Source: PubMed