Evkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol

Core Insights - The FDA has approved Evkeeza (evinacumab-dgnb) for treating children aged 1 to less than 5 years with homozygous familial hypercholesterolemia (HoFH), expanding its indication from previous approvals for older age groups [1][3] - Evkeeza has demonstrated the ability to lower low-density lipoprotein cholesterol (LDL-C) by approximately 50% when used alongside standard lipid-lowering therapies in patients with HoFH [1][2] - The approval highlights the urgent need for effective treatments for HoFH, a severe genetic condition affecting around 1,300 individuals in the U.S. [2][3] Company Overview - Regeneron Pharmaceuticals, Inc. is a leading biotechnology company focused on developing life-transforming medicines for serious diseases, utilizing proprietary technologies like VelocImmune to create fully human monoclonal antibodies [4][16] - The company has a commitment to rare diseases, with ongoing clinical development programs targeting various conditions, including neuromuscular and genetic diseases [4][16] - Regeneron is collaborating with Ultragenyx for the clinical development and commercialization of Evkeeza outside the U.S. [7] Product Information - Evkeeza is an injectable prescription medicine used in conjunction with diet, exercise, and other LDL-C lowering therapies for patients with HoFH aged 1 year and older [9] - The drug works by blocking the function of angiopoietin-like 3 (ANGPTL3), a protein that inhibits lipoprotein lipase and regulates circulating lipids [5][6] - Common adverse reactions reported include nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, and fatigue [3][15] Clinical Data - The extended indication for Evkeeza was supported by clinical efficacy and safety data from a small cohort of 6 children with HoFH, with no new safety concerns identified [3] - The initial approval of Evkeeza was based on a placebo-controlled trial demonstrating significant LDL-C reduction in a high unmet need population [1][2] Patient Support - Regeneron offers the myRARE patient support program to assist patients prescribed Evkeeza, providing financial assistance and resources for accessing the medication [4]