The Rise of ‘Evkiza’: A Breakthrough Treatment for Homozygous Familial Hypercholesterolemia

2023-08-20 04:10:00

[이데일리 김진호 기자]The’Blockbuster Search Part 1’, which took place throughout 2022, dealt with the world’s best-selling pharmaceuticals in turn from 1 to 55 in 2020, three years ago. ‘Blockbuster Exploration Part 2′ discovers and deals with drugs that have newly generated more than $1 billion in sales last year or are expected to generate corresponding sales within 3 to 4 years. We look at the birth process and sales prospects of drugs that have emerged as so-called new blockbusters.[편집자 주]’Evkiza’ (ingredient name: evinacumab), a homozygous familial hypercholesterolemia treatment developed by Regeneron Pharmaceuticals in the US. (Provided by Regeneron Pharmaceuticals)

The marketability of homozygous familial hypercholesterolemia treatment ‘Evkiza’ (ingredient name: evinacumab), approved in April and June 2021 by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), respectively, is significant. is expanding The drug, developed by Regeneron Pharmaceuticals in the U.S., sold $48 million in the U.S. last year, up 153 percent from the previous year ($19 million).

Cholesterol is present in all tissues, including the brain, nerves, muscles, skin, liver, intestines, and heart, and plays an important role in making cell membranes, hormones, and vitamin D. However, if the amount is excessive, it can lead to arteriosclerosis. Hypercholesterolemia is a disease in which cholesterol in the blood is excessive, and can be genetic or acquired (eating habits).

In particular, familial hypercholesterolemia is a chromosome-dominant genetic disease, and ‘low-density lipoprotein cholesterol’ (LDL) receptors are lacking or deleted, resulting in LDL metabolism disorders. The disease occurs in 1 in 500 to 1,000 people worldwide, and it is known that there are about 100,000 patients in Korea. Such patients have very high LDL levels from childhood, and sudden death may occur due to acute coronary ischemia or myocardial infarction around the age of 30.

Among them, homozygous familial hypercholesterolemia, an ultra-rare genetic disease, occurs when one inherits one gene from each parent, and the LDL level is dangerous at 400 mg per 100 ml. Such patients can suffer from early atherosclerotic disease or heart-related diseases as early as their teens.

Ebinacumab, a component of Evkiza, binds to ‘angiopoietin-like protein-3’ (ANGPTL-3) and inactivates it. This allows faster breakdown of fats that lead to high cholesterol when ANGPTL-3 is not functioning properly. Evkiza was listed as the world’s first ANGPTL3 inhibitor-type new drug.

Initially, the FDA and EMA approved EVKIZA as a treatment for homozygous familial hypercholesterolemia in adolescent and adult patients aged 12 years or older. Evkiza is administered by intravenous injection once a month according to body weight at a dose of 15 mg per 1 kg. In the United States, the annual cost of administering Evkiza is known to be about 450,000 dollars (about 500 million won).

As the age of use of Evkiza has recently been extended to include children, sales are expected to grow even more in the second half of this year. In March, the FDA approved the expansion of indications for ‘Evkiza’ as an adjuvant therapy in combination with other cholesterol-lowering drugs for homozygous familial hypercholesterolemia patients aged 5 to 11 years.

At the time, George Yankopoulos, CEO of Regeneron Pharmaceuticals, said, “Since Evkiza, the first ANGPTL-3 inhibitor, obtained approval, it has already established itself as a standard therapy for homozygous familial hypercholesterolemia patients aged 12 years or older.” Patients over the age of 10 will be able to benefit from treatment with Evkiza.”

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