Orphan drug, Juxtapid gets FDA approval for treating HoFH
The US Food and Drug Administration announced the approval of Juxtapid (lomitapide), a new orphan drug used to reduce cholesterol in patients with the rare disorder, homozygous familial hypercholesterolemia (HoFH), according to an FDA news release Wednesday.
The drug is manufactured by Cambridge, Mass.-based Aegerion Pharmaceuticals Inc.
The once-a-day capsule, used in conjunction with a low fat diet and other lipid-lowering treatments, is used to reduce low-density lipoprotein (LDL) cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL) cholesterol in patients with the rare (approximately one in one million Americans) cholesterol disorder.
“Juxtapid, in addition to diet changes and other cholesterol-lowering treatments, is a new option for those suffering with HoFH and the serious health consequences resulting from this condition,” said Eric Colman, M.D., deputy director of the Division of Metabolism and Endocrinology Products in the FDA’s Center for Drug Evaluation and Research.
According to the FDA, HoFH is a rare inherited condition that makes the body unable to remove LDL cholesterol, often called the “bad” cholesterol, from the blood, causing abnormally high levels of circulating LDL cholesterol.
For those with HoFH, heart attacks and death often occur before age 30. Juxtapid works by impairing the creation of the lipid particles that ultimately give rise to LDL.
The most common adverse reactions in the clinical trial included diarrhea, nausea, vomiting, indigestion, and abdominal pain.