Emergent treatments for β-thalassemia and orphan drug legislations

In many countries, β-thalassemia (β-THAL)is not uncommon; however, it qualifies as a rare disease in the USA and in European Union (EU), where thalassemia drugs are eligible for Orphan Drug Designation (ODD). In this review, we evaluate all 28 ODDs for β-THAL granted since 2001 in the USA and the EU: of these, ten have since been discontinued, 12 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.