Evinacumab treatment of pure family hypercholesterricemia (HoFH): FDA accepts biological license application (BLA)

HoFH is an ultra-rare genetic disease affecting about 1,300 patients in the United States.
higher levels of bad cholesterol (low-density lipoprotein cholesterol or LDL-C) in hoFH patients, which increases their risk of early atherosclerosis and heart events.
pharmaceutical company Regeron recently announced that the U.S. FDA has given priority review to evinacumab bio-licensing applications (BLA).
Evinacumab is a research all-human monoclonal antibody that binds and blocks angiogenesin sample 3 (ANGPTL3).
is currently conducting research on Evinacumab's treatment of HoFH (Phase III clinical), incurable hypercholesterlesteremia (Phase II clinical) and severe hyperglyceride triglyceridesemia (Phase II clinical).
study published in NEJM in 2017 by the Regeneron Center for Genetics found that patients with loss of function in the ANPTL3 gene significantly reduced their critical lipid levels, which could reduce LDL-C in HoFH patients by blocking the ANGPTL3 protein.
EU regulation of Evinacumab is also in progress.
2020, the European Medicines Agency's Human Medicines Commission (CHMP) recommended an accelerated evaluation of evinacumab.
Regeneron used the company's VelocImmune ® technology to invent evinacumab, a proprietary genetically engineered mouse platform with a human-generated immune system that produces optimized all-human monoclonal antibodies.
VelocImmune technology has been used to create a variety of FDA-approved antibodies, including Praluent®, Dupixent®, Libtayo ® (cemiplimab-rwlc) and Kevzara® (sarilumab).
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