Dec. 20, 2024 -- The FDA has approved olezarsen, a new drug that helps reduce high triglyceride levels in adults with a rare genetic condition called familial chylomicronemia syndrome (FCS). Marketed under the brand name Tryngolza, the drug is the first treatment for the condition and is to be taken in combination with a low-fat diet.
FCS happens when the body doesn’t have enough lipoprotein lipase, an enzyme essential for breaking down fats (triglycerides) in the blood. Without this enzyme, triglycerides build up, which can lead to inflammation of the pancreas. FCS affects about 3,000 people in the U.S. and can cause symptoms such as recurrent severe stomach pain, tiredness, and acute pancreatitis. People with FCS typically rely solely on strict fat-restricted diet and lifestyle modifications as preventive measures.
Olezarsen blocks a specific protein called apolipoprotein C-III (APOC-III), which is normally responsible for slowing down the breakdown of fats. By lowering APOC-III levels, olezarsen helps the body break down and clear out these fats more effectively, thus lowering triglyceride levels in the blood. Olezarsen (Tryngolza) is expected to be available in the U.S. by the end of the year and is meant to be used once every month as an add-on treatment to low-fat diets (diets with 20 grams of fat or less per day), according to a press release from Ionis Pharmaceuticals, the drug’s maker. It will be provided as an autoinjector, a device designed for use by patients or their caregivers after appropriate training.
The approval followed a successful clinical trial in 66 adult patients with FCS and very high triglyceride levels. Patients received either Tryngolza or a placebo every four weeks for 53 weeks. After six months, triglyceride levels reduced by 42.5% in those taking Tryngolza (80 milligrams) compared with the placebo group. After 12 months of treatment, the reduction increased to 57%. Additionally, Tryngolza helped reduce the number of episodes of acute pancreatitis over 12 months. Only 5% of patients on Tryngolza had an episode, while 30% in the placebo group had one or more episodes.
The most common side effects were skin reactions from the injection, low platelet count, and joint pain. Ionis Pharmaceuticals has warned patients of potential allergic reactions and advised them to inform their doctors of any history of such reactions that had required treatment before starting Tryngolza.