While there are existing therapies for individuals living with homozygous familial hypercholesterolemia (HoFH), a rare form of high LDL cholesterol, these therapies often fail to control cholesterol levels for many patients. But the FDA recently approved Evkeeza (evinacumab-dgnb) as an adjunct therapy for patients with HoFH aged 5+. According to a news release from biotechnology company Regeneron Pharmaceuticals, Inc. (“Regeneron”), Evkeeza, used in conjunction with the standards-of-care, can significantly reduce LDL cholesterol levels.

The drug’s approval hinged on data from the Phase 3 ELIPSE HoFH study, the results of which you can find in the New England Journal of Medicine. Altogether, 65 participants enrolled. During the study, patients received either lipid-lowering therapies by themselves or lipid-lowering therapies in conjunction with Evkeeza. Patients whose treatment regimens included Evkeeza saw their LDL levels decrease by nearly half. Evkeeza patients saw an average LDL reduction of 135 mg/dL compared to just 3 mg/dL on lipid-lowering therapies alone. This reduction was sustained over time. Further, Evkeeza also helped lower non-HDL cholesterol, total cholesterol, and apolipoprotein B. Finally, Evkeeza showed benefits in patients whose HoFH did not previously respond well or at all to treatment.

Although Evkeeza was relatively safe and well-tolerated, some adverse effects did occur. These include flu-like illness, nausea, dizziness, general weakness and lack of energy, pain in the extremities, runny nose, and the common cold. Evkeeza may cause allergic reactions, so if you take Evkeeza and experience inflammation, difficulty breathing, or rashes, please speak to your doctor as soon as possible.

Evkeeza is an angiopoietin-like 3 (ANGPTL3) inhibitor and monoclonal antibody; ANGPTL3 plays a role in the way lipids (fats) are metabolized. It is administered via intravenous infusion once monthly. Evkeeza was developed using Regeneron’s proprietary VelocImmune technology. If you need financial assistance for any out-of-pocket costs related to Evkeeza treatment, or help finding other resources, you may reach out to Regeneron’s myRARE program at 833-385-3392.

What is Homozygous Familial Hypercholesterolemia (HoFH)?

There are two forms of familial hypercholesterolemia: homozygous and heterozygous. Heterozygous is the more common form. Not only is homozygous familial hypercholesterolemia rarer, but it is also more severe. HoFH is a rare inherited form of high cholesterol. APOB or PCSK9 gene mutations are believed to play a role. In people with HoFH, LDL cholesterol levels can rise to more than 4x the normal level; LDL levels may reach 400-1000 mg/dL or more. Angel, who shared her HoFH story in a Patient Worthy interview, mentioned that her levels rose to 1200 mg/dL. Without treatment, HoFH may lead to heart disease or have other negative health impacts. Outside of extremely high cholesterol levels, symptoms of HoFH may include:

• Shortness of breath
• Rapid heartbeat
• Xanthomas and xanthelasmas (yellowish fat deposits on and under the skin)
• Chest pain
• White or gray circles on the cornea
• Swollen or painful Achilles tendon

Treatment options have included apheresis, statins, and ezetimibe.