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Regeneron treatment fights ultra-rare form of high cholesterol in children

Published on 24/05/22 at 09:45am

Regeneron has shared positive results from its Phase III trial of a candidate drug, for children with a rare, life-threatening condition characterised by high cholesterol.

The drug, Evekeeza (evinacumab), demonstrated success in treating elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular disease (ACVD).

The latest Phase III trial results evaluated Evekeeza to treat HoFH, an inherited disease that is also the most severe form of familial hypercholesterolemia. HoFH affects one in 160,000 to 300,000 people worldwide, and around 1,300 people in the US.

Those diagnosed with the disease, including patients as young as teenagers, are at high risk of premature atherosclerotic disease and life-threatening cardiac events.

Evekeeza is a fully human monoclonal antibody that binds to and blocks ANGPTL3, inhibiting lipoprotein lipase and endothelial lipase and regulating LDL-C and other circulating lipids.

In the clinical trial, the drug was tested in kids ages 5 to 11 years old. It met the primary endpoint of reducing the low-density lipoprotein cholesterol by 48% on the 24th week. About 79% of participants saw their LDL-C drop by at least 50% at week 24. All levels of lipid endpoint parameters also saw declines within the first eight weeks.

"Children living with HoFH have an incredibly rare and severe disease that causes dangerously high LDL-C levels. On current treatment options alone, many patients don't reach their treatment goals, leaving them with an uncertain future. Evinacumab has already demonstrated significant LDL-C reductions in adolescents and adults with HoFH. This latest Phase III trial illustrates the potential of this medicine to be a breakthrough HoFH therapy for children as young as five years old, helping them control their LDL-C early in the course of their disease," M Doortje Reijman, MD, the trial's lead investigator, said in a statement. Dr Reijman is also a research associate in pediatric metabolic diseases and nephrology at Amsterdam University Medical Center. 

Lina Adams

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