The FDA has approved the PCSK9 inhibitor alirocumab as adjunctive LDL-c lowering therapy in the treatment of homozygous familial hypercholesterolemia (HoFH). HoFH occurs in approximately 1 in 250,000 individuals and is characterized by very high circulating LDL-c levels ranging from 500-1000 mg/dL. Patients have a high risk developing CVD during adolescence or young adulthood and most do not respond substantially to current LDL-c lowering therapies.

The efficacy and safety of alirocumab was assessed in a 12 week, double-blind, randomized trial among adults with HoFH. Patients were randomized to 150 mg of alirocumab injections (n=45) every 2 weeks or placebo (n=24) in addition to other LDL-c lowering therapies (LLT). The primary outcome was percent change in LDL-c from baseline.

After 12 weeks, patients who were receiving alirocumab and LLT had an average 27% decrease in LDL-c compared to 9% in patients who were on their prescribed LLT.

The common side effects were nasopharyngitis, injection site reactions, and influenza.

Source: U.S. Food and Drug Administration, April 1, 2021