Genzyme and Isis Announce Submission of European Marketing Authorization Application for Mipomersen (Kynamro®)
Submission Seeks Approval for Mipomersen’s Use in the Treatment of Homozygous Familial Hypercholesterolemia and Severe Heterozygous Familial Hypercholesterolemia
CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis Pharmaceuticals Inc. (NASDAQ: ISIS) today 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 U.S. 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 today 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 antisense technology to meet unmet medical needs.”
About Mipomersen
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)).
About 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)).
About Genzyme, a Sanofi Company
One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since its founding in 1981, the company has introduced breakthrough treatments that have provided new hope for patients. The company’s areas of focus are rare genetic diseases, multiple sclerosis, cardiovascular disease, and endocrinology. Genzyme is a Sanofi company. Genzyme’s press releases and other company information are available at genzyme.com.
About Isis Pharmaceuticals, Inc.
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. Isis' drug development programs are focused on treating cardiovascular, metabolic, and severe and rare/neurodegenerative diseases and cancer. Isis' partners are developing antisense drugs invented by Isis to treat a wide variety of diseases. Isis and Alnylam Pharmaceuticals are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development and commercialization of microRNA therapeutics. Isis also has made significant innovations beyond human therapeutics resulting in products that other companies, including Abbott, are commercializing. As an innovator in RNA-based drug discovery and development, Isis has designed and executed a patent strategy that has provided the Company with strong and extensive protection for Isis’ drugs and technology. Additional information about Isis is available at isispharm.com. |
