The Food and Drug Administration (FDA) has approved Praluent (alirocumab) as an adjunct to other low-density lipoprotein cholesterol (LDL-C)-lowering therapies in adults with homozygous familial hypercholesterolemia (HoFH) to reduce LDL-C.
The approval was based on data from the multicenter, double-blind, placebo-controlled phase 3 ODYSSEY HoFH trial (ClinicalTrials.gov: NCT03156621), which assessed the efficacy and safety of alirocumab in adults with HoFH who were taking maximally tolerated doses of statins with or without other LDL-C-lowering therapies (N=69). Patients were randomly assigned to receive either alirocumab 150mg every 2 weeks (n=45) or placebo (n=24). The primary endpoint was the percent change in LDL-C at week 12 from baseline.
Findings showed that treatment with alirocumab resulted in a mean reduction in LDL-C of 27% compared with an increase in LDL-C of 9% for placebo (treatment difference -36%; 95% CI, -51, -20; P <.0001). Alirocumab was also associated with significant mean reductions on other lipid parameters vs placebo, including apolipoprotein B (treatment difference -30%; 95% CI, -42, -17), non-high-density lipoprotein cholesterol (treatment difference -33%; 95% CI, -48, -18), and total cholesterol (treatment difference -27%; 95% CI, -39, -14) (all P <.0001). Patients with 2 LDL-receptor negative alleles (little to no residual function) were reported to have had a minimal to absent response to alirocumab.
The most commonly observed treatment-emergent adverse effects included nasopharyngitis, injection site reactions, and influenza.
Praluent, a proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor, is also approved as an adjunct to diet, alone or in combination with other lipid-lowering therapies (eg, statins, ezetimibe) in adults with primary hyperlipidemia, including heterozygous familial hypercholesterolemia, to reduce LDL-C; and to reduce the risk of myocardial infarction, stroke, and unstable angina requiring hospitalization in adults with established cardiovascular disease.
The product is supplied as 75mg/mL and 150mg/mL in a single-dose pre-filled pen for subcutaneous administration.
1. FDA approves add-on therapy for patients with genetic form of severely high cholesterol. [press release]. Silver Spring, MD: US Food and Drug Administration; April 1, 2021.
2. Blom DJ, Harada-Shiba M, Rubba P, et al. Efficacy and safety of alirocumab in adults with homozygous familial hypercholesterolemia: The ODYSSEY HoFH trial. Journal of the American College of Cardiology. Published online July 14, 2020. doi.org/10.1016/j.jacc.2020.05.027.
3. Praluent [package insert]. Tarrytown, NY: Regeneron Pharmaceuticals, Inc; 2021.