Regeneron Pharmaceuticals, Inc. recently made a significant announcement regarding pediatric healthcare. The U.S. Food & Drug Administration (FDA) has extended the approval of Praluent® (alirocumab) to include children aged 8 and older diagnosed with heterozygous familial hypercholesterolemia (HeFH). This development is an important step forward in addressing the needs of young patients dealing with high levels of LDL-C (bad cholesterol).

Dr. Mary P. McGowan, Chief Medical Officer of the Family Heart Foundation, emphasized the importance of this approval, noting that while many children with HeFH can benefit from existing therapies, there remains a subset for whom LDL-C levels persist dangerously high. Praluent offers an additional option for managing and reducing LDL-C levels early in life.

HeFH is a genetic condition characterized by elevated LDL-C levels due to mutations in genes regulating cholesterol processing. Praluent, already approved for adults with HeFH and homozygous familial hypercholesterolemia (HoFH), has now been extended to include pediatric patients with HeFH.

The FDA's decision was supported by a Phase 3 trial involving pediatric patients aged 8 to 17 with HeFH, already on lipid-lowering medications. Those treated with Praluent every four weeks experienced a significant 31% reduction in LDL-C compared to placebo at 24 weeks, with improvements in other lipid parameters as well. Importantly, the safety profile of Praluent in this trial mirrored that seen in adults with HeFH.

Dr. George D. Yancopoulos, a principal inventor of Praluent, hailed this approval as a testament to Praluent's impact on familial hypercholesterolemia treatment. Praluent's mechanism involves inhibiting PCSK9, thereby increasing LDL receptor availability in liver cells to clear LDL-C from the bloodstream.

Regeneron's VelocImmune technology, utilized in Praluent's development, is a pioneering platform for generating fully human monoclonal antibodies. This technology has been instrumental in creating several FDA-approved therapies, including Praluent, underscoring Regeneron's commitment to innovation in biopharmaceuticals.

Praluent is now indicated for various conditions related to high cholesterol levels in adults and children, expanding treatment options globally. As with any medication, patients should be aware of potential side effects and discuss them with their healthcare providers.

Overall, the extension of Praluent's approval represents a significant advancement in pediatric cardiology, offering hope and improved outcomes for children with HeFH.

 

Source: globenewswire.com

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