Sickle Cell Disease gets its orphan drug by Global Blood in Europe
Global Blood therapeutics recently announced orphan drug designation for its leading product candidate GBT440 .
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.
For Orphan Drug Clinical Insight Reports Contact: email@example.com
Need custom market research solution? We can help you with that too.
Kuick Research is a market research and analytics company that provides targeted information for critical decisions at business, product and
service levels. We are quick, predictive and known by the recommendations we have made in the past. Our result-oriented research methodology offers
understanding of multiple issues in a short period of time and gives us the capability to keep you full with loads of practical ideas.