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FDA Advisory Committee Recommends Approval of Kynamro (Mipomersen) for Familial Hypercholesterolemia
Agency decision expected in January 2013 (Oct. 18)
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee has voted 9 to 6 that sufficient efficacy and safety data have been provided to support the marketing of Kynamro (mipomersen sodium, Genzyme) for the treatment of patients with homozygous familial hypercholesterolemia (HFH). Many people with HFH have aggressive cardiovascular disease beginning in childhood and are at significant risk of cardiovascular events.
The committee's input will be considered by the FDA in its review of the new drug application (NDA) for Kynamro. The FDA is not bound by the committee's guidance, but takes its advice into consideration when reviewing investigational medicines. The drug’s manufacturer, Genzyme, submitted the NDA on March 29, 2012, and the FDA has set a target action date of January 29, 2013.
In considering the benefits and risks associated with Kynamro, the committee reviewed data from one pivotal phase III double-blind, placebo controlled study in HFH patients, three supportive phase III studies in other high-risk hypercholesterolemia populations, and one ongoing long-term extension study.
Patients with familial hypercholesterolemia (FH) develop inherited abnormalities in liver cells that are responsible for clearing low-density lipoprotein (LDL) particles from the blood. FH is autosomal dominant, which means that all first-degree relatives of FH patients have a 50% chance of having the disease as well, making early detection through family screening critically important.
Kynamro is a first-in-class apolipoprotein B (apo B) synthesis inhibitor currently under regulatory review for patients with HFH to further reduce LDL cholesterol (LDL-C) in patients who are on a stable regimen of maximally tolerated lipid-lowering therapies, and who require additional, significant lipid-lowering treatment. The drug is intended to reduce LDL-C by preventing the formation of atherogenic lipoproteins, the particles that carry cholesterol through the bloodstream. Kynamro acts by blocking the production of apo B, the protein that provides the structural core for these atherogenic particles, including LDL and lipoprotein(a).
Source: Sanofi, October 18, 2012.