Aegerion Pharmaceuticals, Inc., a biopharmaceutical company dedicated to the development and commercialisation of novel, life-altering therapies for patients with debilitating, often fatal, rare diseases, has received the US Food & Drug Administration (FDA) approval for Juxtapid (lomitapide) capsules as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B) and non- high-density-lipoprotein cholesterol (non-HDL) in patients with homozygous familial hypercholesterolemia (HoFH).
"We are excited that Juxtapid will become a new treatment option for patients with HoFH," said Marc Beer, chief executive officer at Aegerion. "The approval of our first product also marks an important corporate milestone for Aegerion and reflects our commitment to help patients in need."
HoFH is a serious, rare genetic disease that impairs the function of the receptor responsible for removing LDL-C ("bad" cholesterol) from the body. A loss of LDL receptor function results in extreme elevation of blood cholesterol levels. HoFH patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.
"The FDA approval of Juxtapid is a major step forward for HoFH patients and their families, who have long been waiting for new therapies," said Katherine Wilemon, president and founder of The FH Foundation. "New treatments, combined with further understanding and awareness of this disease, can bring much needed hope to the HoFH community."
Juxtapid contains a Boxed Warning citing the risk of hepatic toxicity. The safety and effectiveness of Juxtapid have not been established in patients with hypercholesterolemia who do not have HoFH. The effect of Juxtapid on cardiovascular morbidity and mortality has not been determined. The safety and effectiveness of Juxtapid have not been established in paediatric patients.
The FDA based its approval of Juxtapid on Aegerion's pivotal phase III study, which evaluated the safety and effectiveness of the medicine to reduce LDL-C levels in 29 adult patients with HoFH. The study was a multinational, single-arm, open-label, 78 week trial that was recently published in the November 2, 2012 online version of the Lancet. In this study, Juxtapid was initiated at 5 mg daily and gradually escalated to doses of 10 mg, 20 mg, 40 mg, up to 60 mg, based on tolerability and acceptable liver enzymes levels.
When added to the existing lipid-lowering therapy of the HoFH patients in the study, Juxtapid significantly reduced LDL-C from a baseline average of 336 mg/dL to 190 mg/dL (40% reduction) at Week 26 in the intent-to-treat population with last observation carried forward for the patients who discontinued prematurely. LDL-C was reduced by an average of 50 percent for the 23 patients who completed the study through Week 26. After Week 26, during the safety phase of the study, adjustments to concomitant lipid-lowering treatments were allowed. Average reductions in LDL-C were sustained during chronic therapy.