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Two recent studies have investigated a new drug called olezarsen, designed to reduce levels of triglycerides in the blood. Triglycerides are a form of blood fat that, when elevated, can increase the risk of heart disease and stroke. The drug was found to significantly lower triglyceride levels and other blood fats associated with disease risk. Produced by Ionis Pharmaceuticals, olezarsen may soon be approved for people with familial chylomicronemia syndrome, a rare condition that results in elevated triglyceride levels and increased risk of acute pancreatitis.

High triglyceride levels are associated with an increased risk of heart disease and stroke. People with familial chylomicronemia syndrome have an even greater risk due to their elevated triglyceride levels. Lifestyle interventions such as diet and exercise can help reduce triglyceride levels, but can be difficult for people with this rare condition who need to follow an extremely low-fat diet. The studies found that olezarsen, an injectable experimental drug, significantly reduced triglyceride levels especially in people with severe hypertriglyceridemia or familial chylomicronemia syndrome, who are likely to benefit most from the medication.

The first study included 154 participants with severe hypertriglyceridemia or moderately elevated levels plus cardiovascular risk. They received monthly olezarsen injections or a placebo, with one group receiving a 50mg dose and another an 80mg dose. Those taking olezarsen saw reductions in triglyceride levels by up to 53.1% compared to the placebo group. The second study recruited 66 people with familial chylomicronemia syndrome who received different doses of olezarsen. The results showed significant reductions in triglyceride levels and acute pancreatitis episodes, indicating potential benefits for this rare disorder.

Experts who were not involved in the studies shared their positive thoughts on the research, highlighting the potential effectiveness of olezarsen in lowering triglyceride levels, especially in patients with severely elevated levels. While additional research is needed to determine the drug’s efficacy in people with moderate-to-high triglycerides, most experts believe that olezarsen will be approved for familial chylomicronemia syndrome. Longer-term studies examining safety and efficacy, particularly in a more diverse population, will be necessary before the drug becomes mainstream.

Currently, there are limited treatment options for people with severely elevated levels of triglycerides, making olezarsen a significant advance in the treatment of familial chylomicronemia syndrome. While the drug has shown promising results in lowering triglyceride levels and other blood fats, further studies are needed to confirm its impact on the risk of cardiovascular disease. Overall, experts believe that olezarsen will be a valuable addition to the treatment of familial chylomicronemia syndrome, addressing the need for an effective drug to manage this rare disorder and prevent life-threatening complications like acute pancreatitis.

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