The CHMP noted that Kynamro was effective at reducing LDL cholesterol levels in patients with homozygous and severe heterozygous familial hypercholesterolaemia.
The ENHANCE study was conducted in 720 patients with heterozygous familial hypercholesterolemia, a genetic disease that can boost untreated cholesterol levels above 300.
In another study of patients with heterozygous familial hypercholesterolemia, a genetic disorder that caused high cholesterol, patients who took REGN727 saw their cholesterol decline between 30% and 65% depending on how the medicine was dosed.
Given the severity and rarity of homozygous FH it is difficult to predict how the committee will vote, but the safety concerns raised by the FDA make it extremely unlikely the drug could receive an expanded indication for heterozygous FH anytime in the near future.