Injectable treatment OK’d as add-on Tx for homozygous familial hypercholesterolemia

WASHINGTON — The FDA approved alirocumab (Praluent) injection for the treatment of homozygous familial hypercholesterolemia (HoFH). Alirocumab is not intended as a stand-alone treatment but is approved for use with other treatments for HoFH, a rare genetic disease that affects about one in 250,000 people.

Patients with the disorder are at a risk for premature cardiovascular disease — such as heart attack and heart disease — when they are teens or young adults. Other cholesterol-lowering drugs are not very effective, and many of these patients die before age 30 without aggressive treatment.

“[Alirocumab] works by stopping the proprotein convertase subtilisin kexin type 9 (PCSK9) protein. PCSK9 controls the number of LDL receptors, which are proteins that help remove LDL-C from the bloodstream and enable the breakdown of fats and cholesterol,” the FDA wrote in a statement. “By blocking PCSK9, [alirocumab] allows faster removal of LDL-C from the blood to lower cholesterol levels.” It is given by subdermal injection every two weeks.

A 12-week, double-blind randomized trial of patients with HoFH pitted alirocumab against placebo, with 45 adults receiving 150 mg of the drug and 24 receiving placebo. The patients were also taking other LDL-C lowering drugs over the course of the study. Change in LDL-C from the beginning of treatment initiation to week 12 was the primary outcome measure. Patients who received alirocumab at the 12-week mark had ,on average, a 27% decrease in LDL-C compared with patients who received placebo, who saw an average increase of LDL-C of 9%.

Side effects of the treatment include nasopharyngitis, injection site reactions, and influenza. There have also been reports of serious hypersensitivity reactions among patients taking the drug.

Alirocumab was first approved in 2015 for the reduction of the risk of “heart attack, stroke, and unstable angina… requring hospitalization in adults with cardiovascular disease.” It is also approved as a stand-alone treatment or along with diet and other therapies for the treatment of primary hyperlipidemia.

Regeneron Pharmaceuticals, Inc, garnered the FDA approval.

Candace Hoffmann, Managing Editor, BreakingMED™

Cat ID: 4

Topic ID: 74,4,730,4,192,925