Epeleuton receives orphan-drug designation for Sickle Cell Disease from the FDA
July 19, 2022Afimmune Confirm Alignment with FDA on Planned Pivotal Trial of Epeleuton in Sickle Cell Disease Patients
January 4, 2023Dublin, Ireland, 19 September 2022: Afimmune, a privately held, clinical stage biopharmaceutical company, today announced that the European Medicines Agency (EMA) has granted orphan drug designation to Epeleuton as an investigational treatment for patients suffering from Sickle Cell Disease.
Orphan-drug designation is granted by the EMA 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 5 people in 10,000 in the EU.
“Following a recent similar orphan drug designation from the FDA, Afimmune is extremely encouraged with this additional validation from the EMA. This places Epeleuton in a strong position globally as a potential first-in-class therapeutic for Sickle Cell Disease” said Dr. David Coughlan, PhD, Afimmune’s Chief Technical Officer.
About 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.
About Epeleuton
Epeleuton is 15-hydroxy eicosapentaenoic acid (15(S)-HEPE) ethyl ester, a novel synthetic fatty acid drug product. Afimmune is prioritising Epeleuton for Sickle Cell Disease due to its superior preclinical efficacy, first-in-class opportunity and a significant reduced regulatory pathway. Epeleuton has already received orphan drug designation for the treatment of sickle cell disease from FDA.
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.
About Afimmune
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.
Contact
[email protected]