US biopharmaceutical company Aegerion Pharmaceuticals (Nasdaq: AEGR) is focused on the development and commercialization of therapies for patients with debilitating rare diseases.

Its therapeutic focus is homozygous familial hypercholesterolemia (HoFH) is the most severe form of familial hypercholesterolemia. HoFH is caused by genetic defects inherited from both parents that affect the function of the low-density lipoprotein (LDL) receptor, the receptor responsible for removing LDL-C cholesterol (bad cholesterol) from the body. HoFH is characterized by extremely high levels of LDL cholesterol (LDL-C). HoFH is typically diagnosed by LDL-C levels, physical exam and family history.

National guidelines recommend LDL-C goals of less than 100 mg/dL (2.6 mmol/L) for patients at high risk for CVD and less than 70 mg/dL (1.8 mmol/L) for patients at very high risk. Its main product, Lojuxta (lomitapide) capsules, are intended as an adjunct to a low fat diet and other lipid lowering medicinal products with or without LDL apheresis in adult patients with HoFH. It is based in Cambridge, MA, USA.