FDA approves Regeneron’s Evkeeza for cholesterol treatment
Regeneron announced that the US Food and Drug Administration (FDA) approved its Evkeeza antibody as a first-of-its-kind treatment for adult and pediatric patients with extremely high low-density lipoprotein cholesterol (LDL-C). Clinical trials demonstrated that Evkeeza significantly reduced LDL-C levels.
The drug is a new therapy for people living with homozygous familial hypercholesterolemia (HoFH). Patients with that condition have two copies of a gene that causes elevated LDL. Present in approximately 1,300 people in the U.S., HoFH results in early onset of dangerous blood vessel blockages and very high cholesterol levels that are hard to control with other drugs.
A fully-human monoclonal antibody, Evkeeza connects to and blocks the function of angiopoietin-like 3 (ANGPTL3), a protein that plays a significant role in lipid metabolism. Approximately 1,300 patients in the US are affected by HoFH, also known as homozygous FH. The ultra-rare hereditary condition occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent. It causes severely high levels (>400 mg/dL) of LDL-C (bad cholesterol), putting patients are at risk for premature atherosclerotic disease and cardiac arrests.
Evkeeza is an injectable prescription medicine used along with other low-density lipoprotein (LDL) lowering medicines in people older than 12 years of age with HoFH. It is not known whether this medicine is safe and effective in people with other causes of high cholesterol; whether it is effective on heart problems such as heart attacks, stroke, or death; or whether it is safe and effective in children with HoFH under 12 years of age.
The drug, which has the generic name evinacumab-dgnb, is injected by a physician into the veins through an intravenous (IV) line over 60 minutes and administered every month (4 weeks). Regeneron’s list price is about $450,000 for a year of treatment. Alternative treatments for the condition include frequent filtering of LDL from the bloodstream, which is expensive and time-consuming.
Evkeeza is the second cholesterol-lowering biological drug Regeneron has developed, according to Healthcare Dive. The first one, Praluent, was not widely accepted after being launched because of its steep price, but price reductions have promoted wider usage. Instead of aiming toward a road range of patients, as Praluent did, is designed for small numbers of very ill patients with rare disease.
There are other methods of lowering LDL. Amryt’s Juxtapid, which gained FDA approval in 2012 for its ability to lower LDL by a median of 50 percent in HoFH patients, can trigger spikes in liver enzymes. Another possible treatment for HoFH patients is apheresis, which consists of filtering the LDL cholesterol out of the bloodstream.
While it is more expensive than other treatments, Evkeeza seems to help HoFH patients to reduce their cholesterol even when tacked on to other therapies. In the clinical trial that supported Evkeeza’s approval (ELIPSE-HoFH) 65 patients with a mean LDL levels of 255 milligrams per deciliter — more than double the normal criteria for healthy levels — were enrolled. After 24 weeks of a monthly infusion of Evkeeza infusion alongside other therapies including Praluent and apheresis, LDL levels dropped an average of 47 percent.
According to Regeneron President George D. Yancopoulos, “We are proud to bring Evkeeza to patients with HoFH, and Regeneron is grateful to the patients and doctors who participated in our trials to make this a reality.”
