Regeneron Pharmaceuticals, Inc. REGN announced that the FDA extended the approval of the cholesterol drug Praluent’s (alirocumab) label.

The regulatory body approved the drug as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies to include pediatric patients aged eight and older with heterozygous familial hypercholesterolemia (HeFH).

The approval is based on a phase III, randomized multi-center trial evaluating pediatric patients aged 8 to 17 with HeFH, who had LDL-C levels of 130mg/dL or greater and were already being treated with lipid-lowering medications. Patients were randomized to receive Praluent or placebo every two or four weeks in two consecutive cohorts.

Patients who received Praluent every four weeks had a 31% lower LDL-C than placebo at 24 weeks. Improvements in additional key lipid parameters were also observed.

Familial hypercholesterolemia (FH) is an inherited condition caused by mutations in one of several genes that control how the body processes cholesterol, which can lead to high levels of LDL-C (bad cholesterol).

FH can come in two forms — HeFH, which develops when one mutated gene is inherited from one parent, and homozygous familial hypercholesterolemia (HoFH), which develops when a mutated gene is inherited from both parents. Praluent is approved to treat both children and adults with HeFH and adults with HoFH.

Praluent is already indicated in the United States to reduce the risk of myocardial infarction, stroke and unstable angina requiring hospitalization in adults with established cardiovascular disease. The drug is indicated as an adjunct to diet, alone or in combination with other low-density LDL-C lowering therapies in adults with primary hyperlipidemia, including HeFH to reduce LDL-C.

Praluent raked in sales of $638.9 million in 2023.

Regeneron’s shares have jumped 28.3% in the past year against the industry’s decline of 4.6%.

 

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The company records net product sales of Praluent in the United States, while partner Sanofi SNY records net product sales of Praluent outside the United States and pays Regeneron a royalty on such sales.

REGN also has a collaboration with Sanofi for the asthma drug Dupixent.

Regeneron’s fourth-quarter sales and earnings beat estimates, but lead drug Eylea’s sales declined year over year in the fourth quarter. Eylea sales were under pressure in 2023 due to competition from Roche’s Vabysmo.

Nevertheless, REGN should benefit from Dupixent’s continued label expansions and solid demand. The approval of Eylea HD is also a great boost, generating incremental revenues for the company. Regeneron’s progress with the oncology portfolio is also impressive.