FDA approves Aegerion's new orphan drug Juxtapid for rare cholesterol disorder

The US Food and Drug Administration has recently approved emerging US biotech firm Aegerion Pharmaceuticals’ (Nasdaq: AEGR) Juxtapid (lomitapide) to reduce low-density lipoprotein (LDL) cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL) cholesterol in patients with homozygous familial hypercholesterolemia (HoFH).

Juxtapid is intended for use in combination with a low fat diet and other lipid-lowering treatments and gained backing from an FDA advisory panel in the fall (The Pharma Letter October 19). Lomitapide may generate at least $300 million in annual sales, said William Tanner, an analyst at Lazard Capital Markets quoted by Bloomberg. The estimate is based on a price of $300,000 a year per patient that is similar to costs of other drugs for diseases with few treatment options, he said. Kimberly Lee, a Think Equity Partners analyst, said peak sales may reach $450 million.

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. In the USA, HoFH occurs in around one in one million individuals. 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.