FDA approves Praluent for homozygous familial hypercholesterolemia.- Regeneron

The FDA has approved Praluent (alirocumab), from Regeneron, as an adjunct to other low-density lipoprotein cholesterol (LDL-C)-lowering therapies in adults with homozygous familial hypercholesterolemia (HoFH) to reduce LDL-C. Approval is based on data from the multicenter, double-blind, placebo-controlled phase III ODYSSEY HoFH trial which measured 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). Data 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. no serious aes, permanent treatment discontinuations or deaths were reported during the double-blind treatment period. during the double-blind treatment period, the ae that occurred in at least 5% of patients, and more commonly with praluent, was diarrhea (7% praluent, 0% placebo). the most commonly observed treatment-emergent adverse effects included nasopharyngitis, injection site reactions, and influenza.>