TARRYTOWN, N.Y. - The U.S. Food & Drug Administration (FDA) has expanded the approval of Regeneron (NASDAQ:REGN) Pharmaceuticals' cholesterol medication, Praluent® (alirocumab), to include children aged 8 and older with heterozygous familial hypercholesterolemia (HeFH), an inherited condition that leads to high levels of low-density lipoprotein cholesterol (LDL-C), commonly known as bad cholesterol. This decision provides a new therapeutic option for pediatric patients with HeFH whose LDL-C levels remain high despite existing treatments.
The FDA's decision is supported by a Phase 3 trial involving pediatric patients aged 8 to 17 with HeFH, who were already on lipid-lowering medications. The study found that patients receiving Praluent every four weeks experienced a 31% reduction in LDL-C levels compared to placebo at the 24-week mark. The trial's outcomes, which also noted improvements in other key lipid parameters, were recently published in the Journal of the American Medical Association Pediatrics.
The safety profile for children in the trial was consistent with that observed in adults with HeFH, with no new adverse reactions identified. Common side effects in previous Praluent trials included injection site reactions and flu-like symptoms.
Praluent, which works by inhibiting the binding of PCSK9 to LDL receptors and increasing the number of available receptors on liver cells to clear LDL from the blood, was initially approved as a treatment for adults with HeFH and homozygous familial hypercholesterolemia (HoFH), as well as a preventative for heart attack, stroke, and unstable angina in adults with cardiovascular disease.
Regeneron, the biotechnology company behind Praluent, developed the drug using its proprietary VelocImmune® technology. The medication is now approved in over 60 countries, including the European Union, Japan, Canada, Switzerland, and Brazil.
This approval marks another milestone for Praluent, which was the first therapy targeting the PCSK9 gene for heart disease to receive FDA approval. The information for this article is based on a press release statement from Regeneron Pharmaceuticals, Inc.
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