According to two studies published in The New England Journal Of Medicine, a new drug called olezarsen is designed to reduce levels of bad fat in the blood, known as triglycerides. Olezarsen, produced by Ionis Pharmaceuticals, also reduced the levels of other blood fats associated with disease risk.
The drug could soon be approved for people with a rare condition called familial chylomicronemia syndrome, who are likely to benefit most from the medication. It is shocking to know that 95% of fat consumed by humans is triglycerides, an essential source of energy. After eating, triglycerides eventually reach the bloodstream. Once there, they travel to the muscles to be used as energy or to liver and fat cells for storage.
Even though triglycerides are vital for health, high levels are associated with an increased risk of heart disease and stroke. When triglycerides are at exceptionally high levels, doctors call it hypertriglyceridemia, which can lead to acute pancreatitis in severe cases. Familial chylomicronemia syndrome is a rare condition that results in elevated triglyceride levels. People living with this condition have an even greater risk of developing acute pancreatitis.
An estimated 1 in 4 people in the United States have high triglyceride levels. Researchers say that some of these individuals respond well to medications like statins, but treatments that have a direct effect on triglycerides are limited.
Lifestyle changes like a healthy diet and regular physical activity with reduced smoking can also help reduce triglyceride levels. Some doctors believe that lifestyle changes are extremely difficult to achieve for some people with familial chylomicronemia syndrome. The first study recruited 154 participants with severe hypertriglyceridemia or moderate hypertriglyceridemia plus elevated cardiovascular risk. They received a monthly olezarsen injection or a placebo. Those receiving olezarsen were further split into two groups: The first group received a 50-milligram (mg) dose, and the second group an 80-mg dose.Compared with placebo, those taking olezarsen reduced their triglyceride levels by 49.3% (50 g group) and 53.1% (80 mg group).They also saw significant reductions in other blood fats linked to cardiovascular risk — namely, APOC3, apolipoprotein B, and non-HDL cholesterol.
The scientists recruited 66 people with familial chylomicronemia syndrome for the second study. Split into three groups, the participants received a placebo, 50 mg of olezarsen every 4 weeks, or 80 mg of olezarsen every 4 weeks. The study ran for 53 weeks. At 6 months, the scientists found that the 80-mg dose significantly reduced triglyceride levels, but the 50-mg dose did not.
While olezarsen may be helpful for people with elevated triglycerides, most experts believe its primary use will be in people with familial chylomicronemia syndrome. Experts also say that there are currently no drugs in the U.S. that are effective in lowering triglyceride levels and reducing the risk of pancreatitis. There is a major need for a drug to treat this rare disorder that causes considerable morbidity.
Journal Reference – Bergmark, B. A., Marston, N. A., Prohaska, T. A., Alexander, V. J., Zimerman, A., Moura, F. A., … Sabatine, M. S. (2024). New England Journal of Medicine. doi:10.1056/nejmoa2402309
