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FDA Approves Expanded Indication for Praluent to Lower LDL-C in Pediatric Patients with HeFH


Regeneron’s PCSK9 inhibitor approved to reduce low-density lipoprotein cholesterol in patients 8 years of age and older with heterozygous familial hypercholesterolemia.

Image credit: waldemarus | stock.adobe.com

Image credit: waldemarus | stock.adobe.com

The FDA has approved an expanded indication for Regeneron’s PCSK9 inhibitor Praluent (alirocumab) to include patients 8 years of age and older for the reduction of low-density lipoprotein cholesterol (LDL-C) in those with heterozygous familial hypercholesterolemia (HeFH).1 In clinical trials, the fully human monoclonal antibody, which binds and inhibits to circulating PCSK9, has been found to lower LDL-C in patients with hyperlipidemia either as a monotherapy or combined with other lipid-lowering therapies (LLTs).2

“Many children with (HeFH) are able to substantially improve their LDL-C (bad cholesterol) with currently available therapies,” said Mary P. McGowan, MD, chief medical officer of the Family Heart Foundation, in a press release. “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.”1

Praluent is also indicated to lower the risk of myocardial infarction, stroke, and unstable angina requiring hospitalization in adult patients with cardiovascular disease; to lower LDL-C, as an adjunct to diet, as a monotherapy or combined with other LLTs in adults with primary hyperlipidemia, including HeFH; and to lower LDL-C, as an adjunct to other LDL-C-lowering therapies, in adults with homozygous familial hypercholesterolemia.

The regulatory action was based on findings from a Phase III, randomized, multicenter trial that included patients aged 8 to 17 years with HeFH and LDL-C levels of 130mg/dL or greater who were already treated with lipid-lowering medications. Investigators sought to include a sample size of 90 patients in order to establish 92% power in identifying mean percent change difference in LDL-C of 30% between Praluent and placebo. The number of participants was increased to 150 to properly evaluate safety and tolerability.

Patients were randomly assigned to receive Praluent (N=101) or placebo (N=52) every two or four weeks in two consecutive cohorts. The trial’s primary endpoint was percent change in LDL-C from baseline to week 24 in each of the cohorts.2

Among patients in the treatment cohort, patients administered Praluent achieved a statistically significant 31% lower LDL-C level compared with placebo in both cohorts at week 24. Investigators also noted improvements in other key lipid parameters.

“The efficacy of [Praluent] reported here in pediatric patients with HeFH is generally consistent with that in adult patients, although the LDL-C reduction in the current study appeared to be lower than reductions previously reported in adults,” the study authors wrote in JAMA Pediatrics.2

In terms of safety, Praluent was generally well tolerated, with a profile consistent with prior findings in pediatric and adult patients with HeFH. The most commonly reported adverse events with Praluent (≥5%) were injection site reactions (7%), influenza (6%), and diarrhea (5%).

“The approval of Praluent for the treatment of high cholesterol was a historic landmark achievement, as it was the first approved therapy targeting the genetically-validated PCSK9 target for heart disease,” George D. Yancopoulos, MD, PhD, Regeneron board co-chair, president and chief scientific officer, said in the release. “Praluent has made a meaningful impact in the treatment of adults with familial hypercholesterolemia, and we are proud that our innovation will now be able to help appropriate children with the heterozygous form of this disease manage their dangerously high levels of LDL-C.”1


1. Praluent® (Alirocumab) Injection Receives FDA Approval to Treat Children With Genetic Form Of High Cholesterol. Regeneron. News release. March 11, 2024. Accessed March 11, 2024. https://investor.regeneron.com/news-releases/news-release-details/praluentr-alirocumab-injection-receives-fda-approval-treat

2. Santos RD, Wiegman A, Caprio S, et al. Alirocumab in Pediatric Patients With Heterozygous Familial Hypercholesterolemia: A Randomized Clinical Trial. JAMA Pediatr. 2024;178(3):283–293. doi:10.1001/jamapediatrics.2023.6477.

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