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Silence Therapeutics blood disorder drug gets EU orphan drug tag

Silence Therapeutics PLC said its SLN124 medicine was granted the orphan drug designation by the European Medicines Agency for treating beta-thalassemia, a blood disorder.

The designation — which grants the therapy expedited clinical development and 10 years of exclusivity if approved — was given by the EMA's Committee for Orphan Medicinal Products.

Beta-thalassemia is an inherited blood disorder that reduces the production of hemoglobin, the protein in red blood cells that transports oxygen throughout the body.

London-based Silence is developing SLN124 for treating iron overload disorders such as beta-thalassemia, myelodysplastic syndrome and hereditary hemochromatosis. Iron overload disorders are characterized by the accumulation of iron in the body.