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FDA grants breakthrough therapy designation to Regeneron for homozygous FH
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Regeneron announced that the FDA has conferred breakthrough therapy designation status on evinacumab for treatment of hypercholesterolemia in patients with homozygous familial hypercholesterolemia.
According to a press release from the company, evinacumab is an investigational monoclonal antibody to the protein ANGPTL3. The protein inhibits lipoprotein lipase and endothelial lipase, and decline in its function has been linked to declines in various lipid parameters.
The company stated that interim phase 2 results for evinacumab in patients with homozygous familial hypercholesterolemia (FH), who have extremely high levels of LDL and often present with signs and symptoms of atherosclerotic CVD before age 20 years, were positive and a phase 3 trial is being planned.
The breakthrough therapy designation is a mechanism to expedite development and review of drugs targeting serious or life-threatening conditions that have demonstrated preliminary clinical evidence of improvement over an existing therapy or placebo, according to the release.