Aegerion Pharmaceuticals, Inc. announced that Japan's Ministry of Health, Labor & Welfare (MHLW) has approved Juxtapid for patients with homozygous familial hypercholesterolemia (HoFH).
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 evaluation of blood cholesterol levels. HoFH patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.
Mary Szela, chief executive officer, Aegerion said, "This approval marks a significant milestone in our ongoing efforts to maximize the value of Juxtapid. Japan is an important market and since lomitapide was granted orphan drug designation for HoFH in 2013, we have worked to establish disease awareness among the HoFH community. We look forward to the opportunity to provide an additional therapeutic option for patients with this severe rare disease. Our team is focused on the upcoming commercial launch, assuming reimbursement approval, and working with Japanese healthcare providers to identify and serve those patients appropriate for Juxtapid therapy."
The MHLW based its approval of Juxtapid on Aegerion's phase III study in Japanese patients, which evaluated the safety and efficacy of the medicine to reduce LDL-C levels in nine patients with HoFH. The findings were consistent with the known safety and efficacy profile of Juxtapid.
Juxtapid is approved in the United States as an adjunct to a low-fat diet and other lipid lowering treatments, including 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-C) in adult patients with HoFH. In the US, Juxtapid carries a boxed warning for the risk of hepatotoxicity. The boxed warning also states that Juxtapid should only be prescribed to patients with a clinical or laboratory diagnosis consistent with HoFH, and that the safety and effectiveness of Juxtapid have not been established in patients with hypercholesterolemia who do not have HoFH. Because of the risk of hepatotoxicity and the importance of Juxtapid only being prescribed to patients with a clinical or laboratory diagnosis consistent with HoFH, Juxtapid is only available through the Juxtapid REMS Programme. The effect of Juxtapid on cardiovascular morbidity and mortality has not been determined.
Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases
