Genzyme and Isis Pharmaceuticals have reported positive Phase III clinical trial results for their cholesterol-controlling drug, mipomersen.
Genzyme is now planning to submit the data from this study to the US Food and Drug Administration for marketing approval of mipomersen.
The drug is designed to block production of a protein that carries LDL cholesterol in the blood. It met its primary goal in a study of 51 patients with a rare genetic abnormality called homozygous familial hypercholesterolemia that increases their cholesterol level.
Patients who took mipomersen for about 26 weeks experienced a 25% reduction in their LDL cholesterol, also called bad cholesterol, as compared to only 3% for patients taking a placebo.
"We are very encouraged by these robust data and the emerging profile of the drug," said Moscicki. "With these results, we remain on track with our development plan for mipomersen."
Besides meeting its main goal of lowering LDL cholesterol, the trial also met each of its three secondary goals of reduction in apolipoprotein B, total cholesterol and non-HDL cholesterol.