(By Balachander) Aegerion Pharmaceuticals Inc. (NASDAQ: AEGR) said the U.S. health regulators have cleared its drug to treat a rare genetic disease that results in extreme elevation of blood cholesterol levels.
The U.S. Food & Drug Administration (FDA) has approved Aegerion's first product JUXTAPID (lomitapide) capsules for the treatment of a rare genetic disease that impairs the function of the receptor responsible for removing bad cholesterol from the body.
The FDA's approval is based on Aegerion's late-stage study which evaluated the safety and effectiveness of the medicine to reduce LDL-C levels in 29 adult patients with homozygous familial hypercholesterolemia (HoFH).
Because of the risk of liver toxicity, JUXTAPID is available only through a restricted program called the JUXTAPID Risk Evaluation and Mitigation Strategy (REMS) Program, Cambridge, Massachusetts-based Aegerion noted.
The company also said safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH.
HoFH patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.
The stock, which has been trading in the 52-week range of $11.75 to $25.74, closed at $25.71 on Friday.