EMA Grants 2 Orphan Designations To Cerenis' CER-001
Biopharmaceutical company, Cerenis Therapeutics, announced that it has received two separate Orphan Drug designations from the European Medicines Agency (EMA) for its drug CER-001 for the treatment of cardiovascular disease.
CER-001, comprised of charged phospholipids, is an engineered complex of recombinant human apoA-I — the major structural protein of HDL. The drug is designed to mimic the natural structure and function of pre-beta HDL. CER-001 plays a role in removing surplus cholesterol and other lipids from tissues, including the arterial wall, where they may cause cardiovascular disease. These lipids are then transported to the liver for disposal via a process labeled Reverse Lipid Transport.
The EMA granted separate Orphan designations to the drug for the treatment of rare genetic defects in HDL synthesis or maturation pathways, namely apoA-I deficiency and ABCA1 deficiency. Inherited defects in either gene can cause Familial Primary HypoAlphalipoproteinemia (FPHA), a rare disease in which HDL particles are absent or severely lacking in the circulation. Absence of HDL compromises the RLT pathway and leads to rapid accumulation of cholesterol in the blood vessels. Patients with FPHA suffer from accelerated atherosclerosis and premature cardiovascular disease.
Dr. John J.P. Kastelein, of the Department of Vascular Medicine at the Academic Medical Center in Amsterdam, The Netherlands, said, “There is no treatment currently available which can directly restore normal HDL levels or normal levels of apoA-I. Despite receiving the best standard of care, many patients have a persistent and extremely high risk of adverse cardiovascular events and premature death, underscoring the unmet medical need for novel therapies. An orphan designation opens up a pathway for CER-001 to be a potential new therapeutic strategy to add to currently available lipid lowering agents to address this elevated risk.”
In June the company reported positive results from two Phase II studies investigating CER-001 in FPHA and Homozygous Familial Hypercholesterolemia (HoFH). The trials met their primary endpoints, showing that CER-001 achieved improvement of RLT in patients. The findings were presented at the European Atherosclerosis Society (EAS) Meetings in Madrid, Spain.
Dr. Jean-Louis Dasseux, CEO of Cerenis, said, “CER-001 has the potential to be an important therapy, not only for patients with rare diseases, such as FPHA, for which there is currently no other therapeutic option, but also for larger patient populations such as post-ACS patients. These two orphan drug designations from the EMA validate our approach and will enable us to accelerate the development of CER-001 and provide this new treatment option to patients.”