Amgen's lipid-lowering drug Repatha receives FDA approval to treat children with HeFH cholesterol disease

CompilationFan Dongdong

A few days ago, the FDA announced that it would approve the use of Amgen’s PCSK9 inhibitor Repatha as an additional treatment for dietary adjustments and statins for the treatment of pediatric patients 10 years of age and older with heterozygous familial hypercholesterolemia (HeFH).

The FDA's approval of Repatha is mainly based on the Phase 3b HAUSER-RCT safety and effectiveness study in children with HeFH aged 10 to 17 years

HeFH is a genetic disease that affects one in every 250 people worldwide

Amgen Repatha is a human monoclonal immunoglobulin G2 (IgG2) that can target PCSK9 in patients and inhibit the binding of circulating PCSK9 to low-density lipoprotein (LDL) receptors (LDLR), thereby preventing PCSK9-mediated The degradation of LDLR allows LDLR to recirculate back to the surface of liver cells, thereby reducing the level of LDL-C in the patient's body

Repatha and Praluent were approved for marketing in 2015, and both focused on PCSK9 protein

However, the two drugs performed mediocre after they went on the market

In the future, as Novartis inclisiran is expected to be approved for this indication as expected, the two drugs may face new competitive threats

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Amgen's Repatha gains an edge on rival Praluent with FDA approval to treat children with rare cholesterol condition