Afimmune’s Epeleuton is granted both FDA Rare Pediatric Disease and Fast Track Designations for the treatment of Sickle Cell Disease
September 18, 2023Afimmune Announces Partnership in Sickle Cell Disease Research with Pathway to Cures
April 19, 2024Afimmune to present at the American Society of Hematology (ASH) Annual Meeting after initiating recruitment in Sickle Cell Disease trial
Dublin, Ireland, 8 November 2023: Afimmune, a clinical stage biopharmaceutical company developing novel rare disease therapeutics, today announced that recruitment has commenced in the first clinical trial evaluating Epeleuton in patients with sickle cell disease (SCD).
The open-label trial design will be presented at the American Society of Hematology (ASH) Annual Meeting to be held in San Diego, California during December 9-12, 2023.
Commenting on the study, Dr. Biree Andemariam, MD, Professor of Medicine and Director of the New England Sickle Cell Institute at UConn Health and study lead investigator said, “This study is an important milestone in the development of a novel therapeutic that may have the potential to address multiple aspects of sickle cell disease. The development and provision of safe and effective therapeutics is an important part of improving outcomes and quality of life for individuals living with sickle cell disease”.
Epeleuton was recently granted Rare Pediatric Disease and Fast-Track designations by the FDA after opening its IND, in addition to orphan drug designations from the FDA and the EMA.
“We are excited to share the progress of Epeleuton’s clinical development with the ASH community. We are working with urgency to bring forward this potential therapy to patients who need effective treatment options” said Dr. Moayed Hamza, Chief Medical Officer of Afimmune.
The accepted abstract is available online on the ASH website:
Session: 114. Sickle Cell Disease
Abstract number: 1159
About Epeleuton
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 the 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.
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
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