Regeneron therapy halves LDL cholesterol levels in rare genetic condition

pharmafile | August 15, 2019 | News story | Research and Development Regeneron, evinacumab, pharma, rare disease 

Regeneron has reported a strong showing for its investigational angiopoietin-like 3 (ANGPTL3) antibody evinacumab in the treatment of the rare genetic condition homozygous familial hypercholesterolemia (HoFH), showing that the therapy met its primary endpoint.

The trial examined the efficacy of evinacumab in patients who, despite treatment with other lipid-lowering therapies including maximally-tolerated statins, PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitors, ezetimibe, LDL apheresis and lomitapide, had an average LDL cholesterol level of 255mg/dl.

It was found that evinacumab in combination with other lipid-lowering therapies reduced LDL cholesterol from baseline by an average of 49% compared to lipid-lowering therapies alone.

Evinacumab was granted Breakthrough Therapy designation by the FDA in 2017 for the treatment of hypercholesterolemia in HoFH patients.

“Currently HoFH patients face limited choices in reducing their LDL cholesterol, including therapies that are time-consuming like LDL apheresis, or that may have side effect concerns,” remarked Dr George D Yancopoulos, President and Chief Scientific Officer of Regeneron. “Despite recent therapeutic advances, there is still a significant unmet need to lower the LDL cholesterol of many patients with HoFH. On average, evinacumab reduced patients’ LDL cholesterol in half and was generally well-tolerated in the trial. These results raise the potential that evinacumab may have value for other patients with severe, refractory hypercholesterolemia, where we have a trial ongoing.”

HoFH affects around 1,300 people in the US. Those living with the condition have severely elevated levels of bad cholesterol (low-density lipoprotein (LDL) cholesterol) which can lead to atherosclerotic disease and cardiac events at even an early age.

Matt Fellows

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