FDA Approves Evkeeza for Youngest Children with Rare Genetic Cholesterol Disorder

Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved Evkeeza (evinacumab-dgnb) for treating children aged 1 to under 5 years with homozygous familial hypercholesterolemia (HoFH), a severe inherited condition causing dangerously high LDL cholesterol levels. This expands Evkeeza’s prior approvals for older children, adolescents, and adults with HoFH.

HoFH, which affects about 1,300 people in the U.S., is caused by inheriting two defective genes that lead to extremely elevated LDL cholesterol from birth, significantly increasing the risk of early heart disease and cardiac events. Many patients remain undiagnosed or are diagnosed late.

The approval was supported by clinical data from six young patients treated under expanded access or compassionate use programs, showing Evkeeza’s safety and effectiveness. Common side effects include nasopharyngitis, flu-like symptoms, dizziness, and fatigue.

Katherine Wilemon, CEO of the Family Heart Foundation, emphasized the importance of this approval for addressing urgent treatment needs in very young children and the critical role of early screening.

Regeneron’s Chief Scientific Officer, Dr. George Yancopoulos, highlighted that this approval reflects the company’s dedication to developing transformative therapies for rare diseases. Regeneron also provides support through its myRARE® program to help patients access Evkeeza.