Praluent (alirocumab) Injection Receives FDA Approval to Treat Children with Genetic Form of High Cholesterol

Approval extends treatment of Praluent to children aged 8 and older with heterozygous familial hypercholesterolemia (HeFH)

TARRYTOWN, N.Y., March 11, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced the U.S. Food & Drug Administration (FDA) has extended the approval of Praluent (alirocumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies to include pediatric patients aged 8 and older with heterozygous familial hypercholesterolemia (HeFH).

“Many children with heterozygous familial hypercholesterolemia (HeFH) are able to substantially improve their LDL-C (bad cholesterol) with currently available therapies. But for those children whose LDL-C remains dangerously high, this approval is an important milestone as it gives these children and their families an additional option to help reduce and manage their LDL-C levels much earlier in their lives,” said Mary P. McGowan, M.D., Chief Medical Officer of the Family Heart Foundation.

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 very 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.

The approval is based on a Phase 3, randomized multicenter 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 (N=101) or placebo (N=52) every two or four weeks in two consecutive cohorts. Patients who received Praluent every four weeks had 31% lower LDL-C than placebo at 24 weeks (97.5% Confidence Interval: -45.0% to -17.9%; p<0.0001). Improvements in additional key lipid parameters were also observed. Results from the trial were recently published in the Journal of the American Medical Association Pediatrics.