Genzyme, a Sanofi company and Isis Pharmaceuticals Inc. announced that Genzyme has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency seeking approval for the 200 mg weekly dose of mipomersen for the treatment of homozygous and severe heterozygous familial hypercholesterolemia.
“This MAA submission is another significant step in the development of mipomersen,” said vice president and general manager of Genzyme’s Cardiovascular Business, Paula Soteropoulos. “We also look forward to our upcoming US regulatory submission later this year, as these submissions move us closer to our goal of making mipomersen available to patients who are in the greatest need of new treatments.”
Genzyme and Isis also announced that, if the necessary approvals are granted, mipomersen would be marketed under the brand name Kynamro, the registered name that has been submitted to health authorities for the investigational agent.
“Mipomersen has the potential to change the management of patients with homozygous and severe heterozygous familial hypercholesterolemia,” said Chairman and CEO of Isis Pharmaceuticals, Stanley T Crooke. “We are excited by the progress we are making with Genzyme on this important development program, which demonstrates the promise of anti-sense technology to meet unmet medical needs.”
Mipomersen is a first-in-class apo-B synthesis inhibitor currently in late-stage development for the reduction of LDL cholesterol (LDL-C). It is intended to reduce LDL-C by preventing the formation of atherogenic lipoproteins, the particles that carry cholesterol through the bloodstream. Mipomersen acts by blocking the production of apolipoprotein B (apoB), the protein that provides the structural core for these atherogenic particles, including LDL and lipoprotein-a (Lp(a)).
Familial Hypercholesterolemia (FH) is a genetic disease that results in elevated LDL-C levels and family patterns of increased risk of premature heart disease and heart disease-related death. FH patients have inherited abnormalities in liver cells that are responsible for clearing LDL particles from the blood. FH is autosomal dominant, which means that all first-degree relatives of FH patients have a 50 percent chance of having the disease as well, making early detection through family screening critically important.
The most severe FH patients have LDL-C levels that are two to four times higher than recommended levels, even when taking multiple cholesterol-lowering medications. These people, who are characterized as having severe FH, include: those who have inherited the disease from both parents (homozygous FH (HoFH)) and those who have inherited it from only one parent, and have a severe form of the disease (severe heterozygous FH (severe HeFH)).
Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases and focusses on rare genetic diseases, multiple sclerosis, cardiovascular disease, and endocrinology.
Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners. The Company has successfully commercialized the world's first antisense drug and has 24 drugs in development.