Amgen PSCK9 monoclonal antibody approved for new indications for pediatric patients over 10 years old

Article source: Medical Rubik's Cube Info

Author: Sunshine

On September 24, Amgen announced that the FDA has approved iloyuumab (trade name: Repatha, Repatha) for the treatment of pediatric patients with heterozygous familial hypercholesterolemia (HeFH) aged 10 years and older.

Familial hypercholesterolemia is an autosomal dominant genetic disease characterized by increased plasma total cholesterol and low-density lipoprotein

PCSK9 (pre-protein converting enzyme subtilisin 9) is a neuronal cell apoptosis-regulating convertase, which participates in liver regeneration, regulates neuronal cell apoptosis, and can also bind to the low-density lipoprotein receptor (LDLR) on the surface of liver cells.

This approval is based on HAUSER-RCT Phase IIIb clinical data

The results showed that compared with placebo, monthly Repatha treatment reduced LDL-C from baseline by 38% (95% CI: 45%, 31%; p <0.

In terms of safety, no new safety signals have been observed

According to the NextPharma database, there are currently 3 PCSK9 inhibitors on the market globally, namely Novartis' inclisiran, Amgen's iloyuumab, and Regeneron/Sanofi's alisiyuumab

In 2020, the global market size of PCSK9 inhibitors is 1.

In China, many companies including Hengrui Pharmaceuticals, Junshi Biologicals, Xinda Biologicals, Kangfang Biologicals, Fuhong Henlius, and Xinlitai have deployed PCSK9 targets

On August 12 this year, Innovent announced that the phase III clinical study (CREDIT-2) of tafolecimab (IBI-306) for the treatment of Chinese heterozygous familial hypercholesterolemia reached its primary endpoint

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