Cerenis Receives EMA Orphan Drug Designations for CER-001 for the Treatment of ApoA-I and ABCA-1 Deficiencies

TOULOUSE, France & ANN ARBOR, Michigan--(BUSINESS WIRE)--Cerenis Therapeutics, the biopharmaceutical company developing CER-001, an engineered human apoA-I-containing pre-beta HDL mimetic, for the treatment of cardiovascular disease, announced today that it has received two separate Orphan Drug Designations from the European Medicines Agency (EMA) for the use of CER-001 in the treatment of patients with rare genetic defects in HDL synthesis/maturation pathways, specifically apoA-I deficiency and ABCA1 deficiency. Inherited defects in the apoA-I gene or the ABCA1 gene in both homozygous and heterozygous forms can act in a dominant manner to cause Familial Primary HypoAlphalipoproteinemia...