FDA Okays New Indication for Alirocumab in Homozygous FH

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The US Food and Drug Administration has approved alirocumab (Praluent, Regeneron Pharmaceuticals) injection as add-on therapy for adults with homozygous familial hypercholesterolemia, the agency announced.

The proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor was originally approved in the United States in 2015 as an adjunct to diet, alone or in combination with other lipid-lowering therapies, to reduce low-density-lipoprotein cholesterol (LDL-C) in adults with primary hyperlipidemia, including heterozygous familial hypercholesterolemia (FH).

Heterozygous FH is one of the most common genetic disorders, affecting one in every 200 to 500 people worldwide, whereas homozygous FH is very rare, affecting about one in 1 million people worldwide.

Alirocumab is also approved to reduce the risk of myocardial infarction, stroke, and unstable angina requiring hospitalization in adults with cardiovascular disease.

The new indication is based on a 12-week randomized trial in 45 adults who received 150 mg alirocumab every 2 weeks and 24 patients who received placebo, both on top of other therapies to reduce LDL-C. At week 12, patients receiving alirocumab had an average 27% decrease in LDL-C compared with an average 9% increase among patients on placebo.

Common side effects of alirocumab are nasopharyngitis, injection site reactions, and influenza. Serious hypersensitivity reactions have occurred among people taking alirocumab.

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