Late-fase data mipomersen bij FH patiënten: 28% LDL-c reductie
Genzyme, Isis present late-stage data on dyslipidaemia drug mipomersen
September 01, 2010
Genzyme and Isis announced Wednesday that results from a late-stage trial of mipomersen in patients with heterozygous familial hypercholesterolemia (heFH) were presented at the European Society of Cardiology Congress. As reported in February, the experimental drug met the main goal of the study, leading to an average 28-percent reduction in LDL cholesterol, compared with an increase of 5 percent among patients who took placebo.
The trial randomised 124 adult patients with heFH to receive mipomersen or placebo weekly for a total of 26 weeks. Data showed that patients treated with mipomersen also experienced a 26-percent reduction in apolipoprotein B, compared with a 7-percent increase for placebo, as well as a 19-percent reduction in total cholesterol, compared with a 4-percent increase for placebo. Further, patients receiving Genzyme and Isis' drug had a 25-percent reduction in non-HDL cholesterol, compared with a 4-percent increase among those given placebo.
Some analysts have expressed concern that the incidence of elevated liver enzymes observed in clinical trials of mipomersen might restrict future sales of the drug. The companies noted that in the trial, 7 percent of patients who received the drug developed elevated liver enzymes, causing three of them to drop out of the study, and six patients treated with mipomersen experienced persistent increases in liver enzymes. Further, patients who received the drug had a median liver fat increase of 4.9 percent, compared with an increase of 0.4 percent for those who took placebo.
Commenting on the data, Paula Soteropoulos, general manager of Genzyme's cardiovascular business, said it "underscore[s] our belief that mipomersen has the potential to help those familial hypercholesterolaemia patients who are 'left behind' by current therapies, and are in need of new treatment options."