Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis Pharmaceuticals Inc. (NASDAQ: ISIS), today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its marketing authorization application (MAA) for KYNAMRO TM (mipomersen) for the treatment of patients with Homozygous Familial Hypercholesterolaemia (HoFH). Genzyme plans to request a re-examination of the CHMP Opinion.
"We are disappointed by the Committee's recommendation. Patients with HoFH carry extreme, ongoing cardiovascular risk with significantly elevated LDL-C levels despite use of currently available therapies,” said David Meeker, President and CEO, Genzyme. “This is a rare disease patient population, with a life-threatening condition, in need of new therapies. We will work closely with the CHMP during the re-examination process to address the Committee's concerns, with the goal of making this important medication available to HoFH patients in Europe."
“We believe that we have generated significant evidence in support of KYNAMRO,” said B. Lynne Parshall, Chief Operating Officer and CFO of Isis. “Patients are in need of new options and we will continue to work with our colleagues at Genzyme toward the marketing approval of KYNAMRO.”
An application for KYNAMRO is currently under review by the U.S. Food and Drug Administration (FDA). In October 2012, KYNAMRO received a positive vote by an FDA advisory panel that Genzyme had provided sufficient efficacy and safety data to support the marketing of KYNAMRO for the treatment of patients with Homozygous Familial Hypercholesterolaemia (HoFH).About KYNAMRO (mipomersen) KYNAMRO is a first-in-class apo-B synthesis inhibitor currently under regulatory review for patients with homozygous familial hypercholesterolaemia (HoFH) to further reduce LDL cholesterol (LDL-C) in patients already maintaining a stable regimen of maximally-tolerated lipid-lowering therapies, and who require additional significant lipid-lowering therapy. It 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 apolipoprotein B (apo B), the protein that provides the structural core for these atherogenic particles, including LDL and lipoprotein-a (Lp(a)).