Sickle Cell Disease gets its orphan drug by Global Blood in Europe

Release Date: 05-Jan-2017

Global Blood therapeutics recently announced orphan drug designation for its leading product candidate GBT440 by European Commission for the treatment of Sickle cell disease (SCD), a severely distressing disease with limited treatment options. The drug is an oral therapy to taken once daily. The action of mechanism for the drug includes increasing the hemoglobin’s affinity for oxygen.


Sickle cell disease (SCD) is an inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, leading to formation of abnormal hemoglobin known as sickle hemoglobin, or HbS. In its deoxygenated state, HbS has a propensity to polymerize, or bind together forming long, rigid rods within RBCs.


European orphan designation is granted to drugs that are intended for the treatment of life-threatening or chronically debilitating rare diseases for which no therapeutic options either exist or are satisfactory. Rare diseases are those defined as having a prevalence of less than 5 in 10,000 in Europe. The designation provides sponsors with development and commercial incentives, including 10 years of market exclusivity, designated product specific consultation by EMA, and certain exemptions from, or reductions in, regulatory fees.



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