Two recent studies have found that an injectable experimental drug called olezarsen significantly reduces levels of triglycerides, which are a form of blood fat linked to increased heart disease risk. These studies, published in The New England Journal of Medicine, looked at the effectiveness of olezarsen in lowering triglyceride levels. The drug, produced by Ionis Pharmaceuticals, also showed reductions in other blood fats associated with disease risk. Individuals with a rare condition called familial chylomicronemia syndrome, which causes elevated triglyceride levels and an increased risk of acute pancreatitis, may potentially benefit the most from this medication.
Triglycerides are a vital source of energy and are present in the fats we eat. However, high levels of triglycerides are associated with an increased risk of heart disease and stroke, especially in cases of severe hypertriglyceridemia. People with familial chylomicronemia syndrome have an even higher risk of developing acute pancreatitis due to elevated triglyceride levels. While treatments like statins can help some individuals lower their triglyceride levels, lifestyle interventions such as diet and exercise may not be as effective for those with genetic factors contributing to their elevated triglyceride levels. The drug olezarsen offers a promising alternative for these individuals.
In the studies, participants with severe hypertriglyceridemia or moderate hypertriglyceridemia plus elevated cardiovascular risk were injected with olezarsen or a placebo. The results showed a significant reduction in triglyceride levels in individuals taking olezarsen compared to those who received a placebo. In another study involving individuals with familial chylomicronemia syndrome, olezarsen was found to reduce triglyceride levels significantly and also showed a reduction in acute pancreatitis cases. Experts believe that olezarsen could soon be approved for individuals with familial chylomicronemia syndrome, providing a much-needed treatment option for this rare disorder.
Experts, including Dr. Cheng-Han Chen and Dr. Gerald Watts, have expressed positive views on the potential benefits of olezarsen in reducing triglyceride levels, especially for individuals with severely elevated levels. While the drug is expected to be approved for familial chylomicronemia syndrome, further research is needed to determine its efficacy for individuals with moderate-to-high triglyceride levels. Longer-term studies focusing on the safety and efficacy of olezarsen, as well as its impact on cardiovascular disease risk, are necessary before this therapy can become mainstream. Additionally, concerns have been raised about the need for more diverse trials to ensure the drug’s effectiveness across different populations.
Overall, the studies on olezarsen show promising results in reducing triglyceride levels, particularly in individuals with familial chylomicronemia syndrome. While the drug may offer significant benefits for this specific group, its potential benefits for individuals with moderately high triglycerides are less clear. Addressing these uncertainties will require further research to establish the long-term safety and efficacy of olezarsen. Despite these considerations, experts believe that the drug could be a valuable addition to the treatment options available for individuals with high triglyceride levels and familial chylomicronemia syndrome.
