Genzyme and Isis Pharmaceuticals have developed a new cholesterol-lowering drug, Mipomersen, which has demonstrated substantial efficacy in Phase III clinical trials but may cause liver problems in 20 percent of patients. Mipomersen is targeted towards patients with a genetic predisposition to elevated blood cholesterol levels (familial
hypercholesterolemia) beginning early in life. These patients are resistant to standard cholesterol-lowering therapies namely statins and prone to developing early-onset atherosclerosis.
While the elevated levels of liver enzymes a sign of liver damage observed in these patients is of some concern, these patients suffer from a potentially life-threatening disease, says ACSH's Dr. Gilbert Ross. So, in comparison to a 20-25 percent rate of elevated liver enzymes, I would say that the benefit to risk ratio shifts towards allowing such patients to take the drug, especially after a comprehensive informed-consent discussion with a physician.
ACSH s Jeff Stier adds, The current FDA focus on safety above all, including benefits, ignores the fact that all drugs have risks. Hopefully, they will take the dire consequences of unchecked lipid levels into account when contemplating approving this new drug.