Sanofi SA's Genzyme And Isis Pharmaceuticals Inc Announce FDA Approval Of KYNAMRO (Mipomersen Sodium) Injection For Treatment Of Homozygous Familial Hypercholesterolemia

Genzyme, a Sanofi SA company and Isis Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for KYNAMROTM (mipomersen sodium) injection. KYNAMRO, given as a 200 mg weekly subcutaneous injection, has been approved as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (Apo B), total cholesterol (TC), and non-high density lipoprotein-cholesterol (non HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH). The FDA approval triggers a $25 million payment to Isis from Genzyme. The FDA approval for KYNAMRO is supported by the clinical trial conducted to-date in the HoFH patient population. The randomized, double-blind, placebo-controlled, multi-center trial enrolled 51 patients age 12 to 53 years, including seven patients age 12 to 16 years, who were maintaining a regimen of maximally-tolerated lipid lowering medications. Treatment with KYNAMRO further reduced LDL-C levels by an average of 113 mg/dL, or 25%, from a treated baseline of 439 mg/dL, and further reduced all measured endpoints for atherogenic particles. KYNAMRO is an antisense drug and is metabolized without affecting the CYP450 pathways used in commonly prescribed drugs, and thus has potential for no drug-drug interactions. No clinically relevant pharmacokinetic interactions were reported between KYNAMRO and warfarin, or between KYNAMRO and simvastatin or ezetimibe.