ESC: Amgen chases pediatric use for cholesterol-busting PCSK9 med Repatha

Amgen's Repatha and Sanofi and Regeneron's Praluent have been locked in a heated battle for supremacy in the pricey PCSK9 cholesterol-cutting market. Looking to angle its way to an advantage, Amgen is looking to adolescent patients with a rare disorder causing high cholesterol levels. 

Adolescent patients with a genetic disorder tied to abnormally high cholesterol treated with Repatha for six months showed a 38.3% relative reduction of LDL-C from baseline compared with placebo, according to late-breaking science presented Saturday at the European Society of Cardiology virtual annual meeting. 

Data from the phase 3 Hauser-RCT trial testing Repatha as an add-on to statins showed Amgen's drug not only reduced cholesterol over placebo but also reduced the levels of another form of cholesterol and cholesterol precursors in the blood, Amgen said.

Heterozygous familial hypercholesterolemia, an inherited condition that causes abnormally high cholesterol, is believed to affect around one in every 250 U.S. adults and adolescents, Amgen said.

Amgen is hoping to expand Repatha from adult patient use—where it received an approval in 2014—to adolescents, a patient group that will come with challenges, according to Chris Kurtz, Amgen's vice president and global development and therapeutic area head for cardiovascular and metabolic. 

For one, many patients often manifest symptoms as adults, so physicians would have to weigh treatment for adolescent patients who have not developed symptoms characteristic of the disorder, Kurtz said. However, earlier treatment could help stave off the development of cardiovascular disease as patients age. 

"We know that people with (this disorder), it’s a disease that only elevates cholesterol," Kurtz said. "It’s certainly plausible that cholesterol elevation early in life contributes to the development of atherosclerosis later in life." 

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