Aegerion Pharmaceuticals Inc. (AEGR) presented new data from its open-label phase III extension study of Juxtapid in patients suffering from homozygous familial hypercholesterolemia (HoFH). This data was presented at the American Heart Association's Scientific Sessions.
The 78-week pivotal study was conducted on 23 patients. Nineteen of them entered the extension phase of the study and continued Juxtapid treatment at their individualized maintenance dose, with 17 completing 126 weeks of therapy.
The primary endpoint of the extension study was mean percent change in low-density lipoprotein cholesterol (LDL-C) from baseline to Week 126. Patients remained on a stable regimen of lipid-lowering therapies during the 26-week efficacy phase of the pivotal study. Mean LDL-C levels were reduced by 45.5% from baseline at week 126.
Juxtapid, indicated for the treatment of patients suffering from HoFH, was launched in the U.S. in late Jan 2013. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency gave a positive opinion on the drug in May 2013 and Aegerion gained EU approval in Jul 2013 under the brand name Lojuxta. Lojuxta will be launched in Europe on a limited basis.
Japan's Ministry of Labour, Health and Welfare granted orphan drug status to Juxtapid for the treatment of HoFH in Sep 2013. The first patient in a therapeutic study is expected to be dosed by year end/early 2014. Aegerion plans to submit a new drug application in Japan in the first half of 2015.
Aegerion carries a Zacks #2 Rank (Buy). Currently, companies like Aeterna Zentaris Inc. (AEZS), Santarus, Inc. (SNTS) and Lannett Company, Inc. (LCI) look more attractive with a Zacks Rank #1 (Strong Buy).