A phase 3 trial into the effectiveness of alirocumab (Praluent) injection has revealed promising results for people with an inherited form of high cholesterol known as heterozygous familial hypercholesterolemia (HeFH) and who require regular weekly or bi-weekly apheresis treatment.
The trial demonstrated that adding alirocumab to existing therapy reduced ‘bad’ cholesterol (low-density lipoprotein cholesterol (LDL-C)) by about 50% from baseline compared to a 2% for placebo. Alirocumab significantly reduced the need for apheresis treatment by 75% compared to placebo, the primary endpoint of the study. The product was developed jointly by Sanofi and Regeneron.
Dr Handrean Soran, Consultant Physician and Endocrinologist at Central Manchester University Hospitals, said: “Apheresis therapy is an invasive, time-consuming and expensive treatment, therefore it is promising to see that alirocumab has the potential to reduce the frequency of this burdensome treatment in patients with inherited high cholesterol.
“These data are particularly exciting as 93% of patients treated with alirocumab experience at least a 50% reduction in their apheresis therapy sessions.”
Despite being treated with apheresis and entering ODYSSEY ESCAPE with very high ‘bad’ cholesterol levels (mean of 4.7mmol/L or 181mg/dL across all treatment arms), 63% of patients treated with alirocumab no longer required apheresis therapy after 6 weeks of receiving alirocumab. At this same time point, the average ‘bad’ cholesterol level among the alirocumab-treated group was 2.3mmol/L (90mg/dL), compared to 4.8mmol/L (185mg/dL) in the placebo group. The recommended target cholesterol level is less than 3mmol/L for ‘bad’ cholesterol, depending on cardiovascular risk.
Apheresis is a procedure similar to kidney dialysis where ‘bad’ cholesterol is removed from the blood, and is usually reserved for high-risk patients with very high cholesterol levels who are unable to achieve their cholesterol-lowering goals on any other therapy. Treatment can cost between £17,000 to £31,000 for each patient per year in the UK. About 200 people in the UK are eligible for the treatment, yet only 30-40 patients receive the treatment each year. Furthermore, there are only 8 apheresis centres in the UK and many patients have to travel for the procedure.
Dr Tunde Falode, Director of the Cardiovascular Division at Sanofi, said: “At Sanofi we are committed to improving the quality of life for people with inherited high cholesterol. We’re therefore extremely encouraged by the results from the ODYSSEY ESCAPE trial, which provide an important insight into the effect of alirocumab when added to existing therapies.
“This is the first clinical trial to demonstrate that alirocumab reduced the frequency of apheresis therapy and these data reinforce the value of alirocumab for patients with high cholesterol.”
Alirocumab received approval from the National Institute for Health and Care Excellence in June and from the Scottish Medicines Consortium in August as a treatment option for people who have raised levels of ‘bad’ cholesterol and who are at a high risk of a heart attack or stroke.