Dublin, Ireland, 19 July 2022: Afimmune, a privately held, clinical stage biopharmaceutical company, today announced that the Food and Drug Administration (FDA) in the United States (US) has granted orphan drug designation to Epeleuton as an investigational treatment for patients suffering from Sickle Cell Disease.
Sickle Cell Disease is a group of inherited, progressive blood disorders carried by the β allele of the hemoglobin gene with an expected 30-year reduced life expectancy. The disease is characterized by abnormal polymerization of hemoglobin during oxygenation which results in the sickling of red blood cells. The disease is rare, with an estimated prevalence of only ~100,000 people affected in the US and ~52,000 people in the EU.
Orphan-drug designation is granted by the FDA to drugs which are defined as those intended for the safe and effective treatment or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States.
“New therapies that meaningfully improve clinical outcomes across all age groups affected by sickle cell disease are urgently needed and Epeleuton, a Phase 2 ready oral asset, is well positioned to meet this unmet medical need” said Dr. Markus Weissbach, MD, PhD, Afimmune’s Chief Medical Officer. “We are extremely encouraged with this validation from the FDA and excited by the therapeutic potential of Epeleuton in Sickle Cell Disease.
Epeleuton is 15-hydroxy eicosapentaenoic acid (15(S)-HEPE) ethyl ester, a novel synthetic fatty acid drug product. Epeleuton has been studied in exploratory clinical trials in different indications. Afimmune is prioritising Epeleuton for Sickle Cell Disease due to the superior preclinical efficacy, first-in-class opportunity and the significantly reduced regulatory pathway.
Epeleuton has been shown to have a unique mechanism of action for the treatment of Sickle Cell Disease, targeting factors affecting severity, course of disease and vaso-occlusive crisis risk. Epeleuton is a Phase 2 ready oral asset. Epeleuton Sickle Cell data were recently presented at the European Hematology Association (EHA) and further information may be found here.
Afimmune, headquartered in Dublin, Ireland, is a clinical stage drug discovery and development company working on new medicines to improve the quality of life for people with rare and inflammatory diseases.