The US Food and Drug Administration (FDA) has approved alirocumab (Praluent) for the treatment of homogeneous familial hypercholesterolemia (HoFH) in adult patients.
The indication, given to Regeneron Pharmaceuticals, gives patients the rare, genetic condition, PCSK9 inhibitor therapy associated with significant reductions in low-density lipoprotein (LDL-C) cardiovascular cholesterol.
Alirocumab is a PCSK9 inhibitor therapy that has been shown to help eliminate LDL-C from the bloodstream of treated patients and allows better distribution of fat and cholesterol. Praluent was approved by the FDA as an injection every two weeks.
The agent was originally approved to reduce the risk of heart attack, stroke and unstable angina – or serious cardiovascular events (MACE) – in adults with cardiovascular disease in 2015. Since then, it has been additionally indicated for the treatment of single or superfluous primary.
The homogeneous form of hypercholesterolemia is characterized by a pair of genetic mutations that alter the body’s ability to clear cholesterol. The primary result is extremely high LDL-C measurements, ranging from 500-1000 mg / dL per patient – a fourfold increase to normal levels.
HoFH is associated with premature cardiovascular disease and MACE risk in adolescent and young adult patients. Death by the age of 30 is a significant risk for patients undergoing treatment.
Currently, 1 in 250,000 people is estimated to have HoFH.
Alirocumab approval for HoFH was based on findings from a 12-week, double-blind, randomized trial, compared with 45 patients receiving 150 mg every 2 weeks and 24 patients receiving placebo.
All test participants received additional LDL-C reduction therapies during the trial.
The researchers said that the primary effect of the percentage change in LDL-C from onset to week 12. At the end of the trial, patients treated with alirocumab reported an average reduction of 27% in LDL-C, while patients receiving placebo reported an average reduction of 9%.
Commonly reported side effects with alirocumab included rhinopharyngitis, injection site reactions, and influenza.