Familial hypercholesterolemia (FH) is a genetic disease that produces very high levels of LDL cholesterol, and and premature coronary artery disease. FH is especially severe when the gene for FH is inherited from both parents, instead of just one - a condition referred to as homozygous FH.
Patients with homozygous FH have extremely high cholesterol levels, and often develop cardiovascular disease at a very young age. Their response to traditional cholesterol therapy (such as statins) is often insufficient. Some of them need to have regular treatments with apheresis (a procedure similar to hemodialysis) to lower their cholesterol.
Last week the Food and Drug Administration approved a new drug, called lomitapide (Juxtapid) for patients with homozygous FH. Lomitapide will be available only through a special program, by the direction of the FDA. These special precautions are necessary because the drug has been shown to produce liver disease in some patients. In fact, given the drug's propensity toward liver toxicity, the only reason the FDA approved it is that lomitapide offers a new therapeutic option to patients with this dangerous and difficult-to-treat condition. Without effective treatment, the risk of serious cardiovascular disease is very high in patients with homozygous FH.
Under the auspices of the FDA, the manufacturer of lomitapide (Aegerion Pharmaceuticals) has opened a support service for patients with homozygous FH and their families, which can help them determine whether this treatment is a reasonable consideration. The toll-free phone number for this new program is 1-85JUXTAPID (1-855-898-2743).