FDA Approves Extended Indication for Evkeeza

Regeneron Pharmaceuticals announced today the extended FDA approval of Evkeeza (evinacumab-dgnb) for the treatment of homozygous familial hypercholesterolemia (HoFH) in patients from age one to less than 5 years old, according to a news release.

Evkeeza is an ANGPTL3 antibody inhibitor, initially approved by the FDA in 2021 for adults and pediatric HoFH patients ages 12 and older. In 2023, the indication expanded to include children as young as 5 years old. It works by binding to and blocking the function of angiopoietin-like 3, a protein that regulates circulating lipids.

HoFH is the most severe form of familial hypercholesterolemia (FH), a genetic disorder that impacts how the body recycles low-density lipoprotein cholesterol (LDL-C), sometimes referred to as “bad cholesterol.” It affects approximately 1,300 people in the United States.

HoFH is inherited from both parents, compared with FH, in which only one parent must be a carrier.

It is diagnosed when LDL cholesterol levels are greater than 400 mg/dl. Additional symptoms can include yellowish skin under the eyes, around the knuckles and around the Achilles tendons, caused by cholesterol deposits. A white, grey or yellow ring may also appear around the cornea.

If left untreated, HoFH can lead to heart disease by age 10, or as early as age 2 or 3. Patients are at risk for both stroke and heart attack.

“The approval of Evkeeza for children as young as 1 year of age addresses a critical unmet need for those with homozygous familial hypercholesterolemia, a life-threatening condition that causes extraordinarily high LDL-C levels from birth,” Katherine Wilemon, founder and CEO of the Family Heart Foundation, said in the news release. “Families and their medical teams will now have an effective treatment option for these very young children who are at risk of serious complications from diseased arteries and calcified valves without timely and sufficient LDL-C lowering. This development underscores the importance of, and urgency needed in, identifying children with FH through pediatric screenings in accordance with guidelines.”

The recommended dosage of Evkeeza is 15 mg administered by intravenous infusion over 60 minutes every four weeks, according to the prescribing information. It should be diluted depending on patient weight, with 15 mg diluted in a maximum of 250 mL for a patient who weighs at least 99 lbs.

The extended indication approval was made using efficacy and safety data from a trial of 6 children with HoFH. The median age of participants was 3 years old with an average LDL-C count of 499 mg/dL. Participants had either taken part in the United States expanded access program or the United States compassionate use program. Patients who received 15 mg of Evkeeza every four weeks for up to 98 weeks showed a mean reduction in LDL-C of 60%.

The most common adverse reactions, which occurred in at least 5% of patients, included nasopharyngitis, influenza-like illness and dizziness. No new safety concerns were identified.

Support for patients prescribed Evkeeza is available through the myRARE patient support program, which offers financial assistance to eligible patients, product information, insurance benefit details and appointment reminders.

The current list price of Evkeeza is $12,574.23 for 2.3 mL.