CSL Behring has announced that its new plasma-derived therapy for Sickle Cell Disease (SCD), CSL889, has been granted the orphan drug designation by, both, the US Food and Drug Administration (FDA) and the European Commission.
The bio-therapeutics giant – which distributes plasma-based therapies across 100 countries for a variety of conditions from Primary Immunodeficiency Disorder (PID) and respiratory diseases to burn and shock treatment – announced that its investigational therapy, based on the protein hemopexin, received the special status on November 2.
The status is special since it is granted to only those therapies intended to treat rare diseases: Diseases, in the US, which affect less than 200,000; and in the European Union, diseases which affect not more than 5 in 10,000 people.
Hemopexin is a crucial protein found in our plasma, the lack of which is highly correlated with symptoms of SCD, particularly Vaso-Occlusive Crises (VOC): episodes of excruciating pain.
Sickle Cell Disease is a hereditary blood disorder wherein the presence of an abnormal kind of hemoglobin causes red blood cells to become crescent or sickle-shaped, instead of round, pillow-shaped. As a consequence, their passage through narrow veins is hindered, which prevents blood from reaching important areas of the body. The phenomenon results in tissue damage, VOC, and even heart disease.
SCD has no cure and its patients are prescribed pain killers to manage VOC. Hence, if its efficacy is clinically proven, CSL's hemopexin-based therapy will represent a major breakthrough in medical science. A landmark innovation.
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