Breakthrough status for a new drug to treat familial hypercholesterolemia.
An experimental monoclonal antibody drug to treat a rare form of inherited high cholesterol received breakthrough therapy designation from federal regulators.
Regeneron announced that the US Food and Drug Administration (FDA) has given that status to evinacumab to lower cholesterol in patients with Homozygous Familial Hypercholesterolemia (HoFH).
The drug inhibits angiopoietin-like protein 3 (ANGPTL3). ANGPTL3 acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism, the company said.
Among current treatments for HoFH are cholesterol-lowering PCSK9 inhibitors. Regeneron makes one of those--alirocumab (Praluent)--and is currently engaged in a patent dispute with Amgen, which makes a rival PCSK9 drug in evolocumab (Repatha).
Regeneron previously reported positive interim phase 2 results for evinacumab in HoFH patients and is currently planning a phase 3 trial.
HoFH is the most severe form of hypercholesterolemia. While rare, occurring in approximately 1 to 2 people per million, untreated patients can have LDL cholesterol levels ranging from 500 to 1000 mg/dL, compared to normal LDL cholesterol levels of less than 130 mg/dL. Due to these high levels of LDL cholesterol, patients with HoFH are at an extreme risk of premature cardiovascular disease. Without treatment, patients typically present with signs and symptoms of atherosclerotic cardiovascular disease before the age of 20.
In its phase 2 trial involving a small group of HOFH patients and controls got the drug for 4 weeks. The mean reduction in LDL-C in the patients who got the drug was 55% at the primary endpoint.
The company reported the new designation in a news release.
Regeneron’s phase 2 results are reported here.