- The FDA has accepted Regeneron Pharmaceuticals Inc's REGN supplemental marketing application seeking approval for Evkeeza (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies for children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH).
- The FDA target action date is March 30, 2023.
- HoFH is an ultra-rare inherited condition that occurs when two copies of the FH-causing genes are inherited, resulting in dangerously high levels of low-density lipoprotein-cholesterol (LDL-C or bad cholesterol).
- The application is supported by data from a three-part trial evaluating Evkeeza in children aged 5 to 11 years with HoFH.
- The trial met its primary endpoint, showing that on average, children who added Evkeeza to other lipid-lowering therapies reduced their LDL-C by 48% at week 24.
- Furthermore, 79% (n=11) saw their LDL-C reduced by at least half at 24 weeks following Evkeeza treatment, with an average absolute reduction in LDL-C from baseline of 132 mg/dL.
- Evkeeza is the first angiopoietin-like 3 (ANGPTL3) targeted therapy approved by the FDA, European Commission, and the U.K.'s Medicines and Healthcare products Regulatory Agency as an adjunct therapy for patients aged 12 years and older with HoFH.
- Price Action: REGN shares are up 0.23% at $737.75 on the last check Wednesday.
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