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 (FPHA), a rare syndrome characterised by the absence of or a severe deficiency of HDL particles in the circulation. Due to either the impaired production/maturation or the premature destruction of HDL particles, the Reverse Lipid Transport (RLT) pathway, the body’s only natural mechanism for the elimination of cholesterol, is compromised. Patients experience a rapid accumulation of cholesterol, particularly in blood vessels, which often results in accelerated atherosclerosis and premature cardiovascular disease.
John J.P. Kastelein, MD, PhD, of the Department of Vascular Medicine at the Academic Medical Center in Amsterdam,