Dublin, Ireland, 18 September 2023: Afimmune, a clinical stage biopharmaceutical company developing novel rare disease therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease and Fast Track designations to Afimmune’s Epeleuton for the treatment of Sickle Cell Disease (SCD).
As a result of the Rare Pediatric Disease designation, once Epeleuton receives approval for SCD from the FDA, Afimmune may qualify for a priority review voucher. This valuable voucher could be redeemed to receive an expedited review of a subsequent marketing application for a different product.
The Fast Track designation enables Afimmune to have more frequent meetings and written communication with the FDA to support a potential shortening of the Epeleuton review process.
Commenting on the update, Ms. Linda Connolly, M.Pharm, Associate Director of Regulatory Affairs of Afimmune said, “These awards are an important acknowledgement by the FDA of the potential Epeleuton holds for patients suffering from Sickle Cell Disease. They also build on the Orphan Drug designations received from both the FDA and EMA. We will continue to work closely and collaboratively with all the key regulators to help bring Epeleuton to affected patients as quickly as possible.”
Epeleuton is 15-hydroxy eicosapentaenoic acid (15(S)-HEPE) ethyl ester, a novel synthetic fatty acid drug product. Epeleuton has been shown to have a unique dual mechanism of action for the treatment of SCD, targeting factors affecting severity, course of disease, and vaso-occlusive crisis risk.
Afimmune is developing Epeleuton for SCD due to its novel disease-modifying preclinical efficacy, first-in-class opportunity and a significantly reduced regulatory pathway. Epeleuton has received orphan drug designation for the treatment of SCD from the FDA and EMA, in addition to rare pediatric disease and fast track designations from the FDA.
About Sickle Cell Disease
SCD 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.
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.