The FDA approval extends Evkeeza’s indication to include children aged five to 11 years with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition characterised by extremely high low-density lipoprotein cholesterol (LDL-C)


Regeneron Pharmaceuticals Headquarters. (Credit: ajay_suresh/ Wikimedia Commons)

Regeneron Pharmaceuticals has received expanded US Food and Drug Administration (FDA) approval for Evkeeza (evinacumab-dgnb) to treat an ultra-rare genetic condition in young children.

Evkeeza is a fully human monoclonal antibody that blocks the function of angiopoietin-like 3 (ANGPTL3), created using the company’s VelocImmune technology.

ANGPTL3 is a protein that inhibits lipoprotein lipase (LPL) and endothelial lipase (EL) and regulates circulating lipids, including low-density lipoprotein cholesterol (LDL-C).

The drug was initially approved in the US, as an adjunct to other lipid-lowering therapies, to treat homozygous familial hypercholesterolemia (HoFH) in people aged 12 years and above.

The current FDA approval expands its indication to include children as young as five years old, to control very high levels of LDL-C caused by HoFH.

Regeneron president and chief scientific officer George D Yancopoulos said: “Since it was first approved, Evkeeza has become the standard of care for homozygous familial hypercholesterolemia in those aged 12 years or older.

“We’re gratified that now children as young as five years old have the potential to benefit from this treatment. As a first-in-class medicine for this relentless disease, Evkeeza exemplifies the promise of genetics-based research to transform treatment paradigms.

“Evkeeza’s journey from target discovery to treatment innovation was only made possible due to our long-term investment in genetics research and monoclonal antibody technologies, and this remains a central tenet of our science-first approach to this day.”

HoFH is an ultra-rare genetic disorder that occurs when two copies of the FH-causing genes are inherited, one from each parent, resulting in critically high levels of LDL-C.

Regeneron said that people living with HoFH are at risk of developing premature atherosclerotic disease and cardiac events even in their teenage years.

The US FDA has granted a Priority Review for the supplemental biologics license for Evkeeza.

The approval is based on a three-part, single-arm, open-label trial that evaluated Evkeeza, when used together with other lipid-lowering therapies in children with HoFH.

In the study, the patients who received the drug, together with other lipid-lowering drugs, experienced a 48% reduction in LDL-C at week 24.

Evkeeza showed a safety profile that was consistent with the safety profile observed in adults and paediatric patients aged 12 years and older.

The most common adverse events (AEs) are headache, oropharyngeal pain, upper abdominal pain, diarrhoea, vomiting, fatigue, nasopharyngitis, rhinitis and cough.

Furthermore, Regeneron is offering myRARE patient support programmes to help eligible patients who need help with the out-of-pocket cost of Evkeeza.