FDA approves alirocumab to treat HeFH patients aged 8 years and up
The approval is based on data from a phase 3, multicenter, randomized trial that evaluated patients aged 8 to 17 years with HeFH who had LDL-C levels of 130 mg / dL or greater who were being treated with lipid-lowering medications.
FDA approves alirocumab to treat HeFH patients aged 8 years and up | Image Credit: © Calin - © Calin - stock.adobe.com.
The FDA has approved an expanded indication for alirocumab (Praluent; Regeneron Pharmaceuticals) for use as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies in patients aged 8 years and older with heterozygous familial hypercholesterolemia (HeFH), according to a press release from Regeneron.
The inherited condition is the result of mutations in 1 of multiple genes that control how the body processes cholesterol, leading to high levels of LDL-C.
The expanded indication approval is based on data from a phase 3, multicenter, randomized trial that evaluated patients aged 8 to 17 years with HeFH who had LDL-C levels of 130 mg / dL or greater who were being treated with lipid-lowering medications.
The primary endpoint of the pediatric trial was the percent change in LDL-C from baseline to week 24 in the alirocumab- and placebo-treated groups. Patients were on a low-fat diet and were being treated with background lipid-lowering medications at baseline.
In the trial, 101 patients received alirocumab and 52 received placebo ever 2 or 4 weeks in a pair of consecutive cohorts. For patients who received alirocumab every 4 weeks, their respective LDL-C was 31% lower compared to placebo at 24 weeks (97.5% CI: –45.0% to –17.9% [P < .0001]).
"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, Family Heart Foundation in the 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."
The safety profile of alirocumab was consistent with the safety profile observed in adults with HeFH, and no new adverse reactions were identified in the trial.
Across alirocumab trials in patients with primary hyperlipidemia (n = 2476), injection site reactions (7%), influenza (6%) , and diarrhea (5%) were the most common adverse reactions (≥5%) more frequently associated with alirocumab compared to placebo.
Reference:
Praluent (alirocumab) injection receives FDA approval to treat children with genetic form of high cholesterol. Regeneron Pharmaceuticals. Press release. March 11, 2024. Accessed March 12, 2024. https://www.biospace.com/article/releases/praluent-alirocumab-injection-receives-fda-approval-to-treat-children-with-genetic-form-of-high-cholesterol/
