The Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to evinacumab (Regeneron) for the treatment of hypercholesterolemia in patients with homozygous familial hypercholesterolemia (HoFH). 

Regeneron previously reported positive interim Phase 2 results which showed that evinacumab added to current lipid-lowering therapy reduced low-density lipoprotein cholesterol (LDL-C) levels by an additional 55% (mean reduction; range 25% to 90%) at week 4 compared to baseline in HoFH patients. The most common drug-related adverse events were injection-site reactions, which were mild in severity. The company is currently planning a Phase 3 trial. 

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Evinacumab is an investigational monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3). ANGPTL3 acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism.

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