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.