ORLANDO, Florida (Reuters) - An experimental drug for patients genetically disposed to extreme high cholesterol being developed by Isis Pharmaceuticals Inc and Genzyme Corp significantly lowered cholesterol in a pivotal late-stage study, according to data presented on Tuesday.
The drug, mipomersen, succeeded in attaining the main goal of the trial, cutting “bad” LDL cholesterol by 25 percent compared with just a 3 percent reduction from placebo in patients with a very rare condition known as homozygous familial hypercholesterolemia.
Genzyme, which will market the drug under a deal with Isis, plans to apply for U.S. and European approval by mid-2011.
People with the condition, which is inherited from both parents, tend to have cholesterol at four times normal levels and are at extreme high risk of heart attack and early death.
Mipomersen also succeeded in all the secondary and tertiary goals of the trial by cutting levels of other dangerous particles, suggesting the drug may offer benefits to patients beyond LDL reduction, researchers said.
Reporting by Bill Berkrot and Ransdell Pierson, editing by Gerald E. McCormick