FDA Approves Tryngolza, First Treatment to Reduce Acute Pancreatitis Risk in Severe Hypertriglyceridemia

What Is It, and Why Does It Matter?

Tryngolza (olezarsen) is a once-monthly injection that is now approved by the FDA to help lower triglycerides and decrease the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG). Before this approval, this medicine was only available for a rare genetic condition called familial chylomicronemia syndrome (FCS). Triglycerides are fats found in your blood. When you have sHTG, your triglyceride levels are extremely high – at least 500 mg/dL. To put this in perspective, healthy triglyceride levels are below 150 mg/dL. To lower their triglyceride levels, people with this condition often take medications and make changes to their lifestyle, including limiting alcohol and eating a low-fat diet. Although other medicines are approved to help lower triglycerides in people with sHTG, this is the first medicine that is also approved to reduce your risk of acute pancreatitis. Acute pancreatitis happens when your pancreas suddenly becomes inflamed. This causes intense stomach pain and often requires you to stay in the hospital. It can be life-threatening. Preventing the first episode is critical because having one attack raises your chances of having more in the future. Tryngolza works by lowering the amount of a protein in your body called apolipoprotein C-III (apoC-III). Reducing apoC-III levels allows your body to break down the triglycerides in your blood and lower your total triglyceride levels.

Why Was It Approved?

The FDA based its approval on findings from two studies involving 1,061 adults with sHTG. People in the studies started with average triglyceride levels around 1,116 mg/dL even though they were already taking standard treatments. Each person was randomly given either Tryngolza 50 mg, Tryngolza 80 mg, or a placebo (an inactive treatment) once every four weeks for 12 months. After six months, triglyceride levels decreased by about 49%-63% in people taking the 50-mg dose, compared with the placebo. In people taking the 80-mg dose, triglyceride levels decreased by about 55%-72% when compared with the placebo. These improvements lasted through the entire 12 months of the study. About 86% of people using Tryngolza reached triglyceride levels below 500 mg/dL, which is an important target for lowering pancreatitis risk. And people who were using the medicine had fewer acute pancreatitis episodes during the study. The most frequent side effect was a reaction – such as redness, itching, a rash, or pain – at the site where the medicine was injected. Liver enzyme levels increased in about 3%-4% of people.

What Do I Need to Know?

Tryngolza is given as a shot under the skin (subcutaneous) once a month. You may give these shots to yourself, or they may be given by a caregiver. After you start using this medicine, your health care provider will monitor your triglyceride levels to find out if you should increase your dose. You should keep following a low-fat diet while you are using this medicine. Before you start this medicine, tell your health care provider about your medical history, all of your medical conditions, and any medicines you are taking. Some people may have an allergic reaction to Tryngolza that can include a hard time breathing, skin redness, hives, face swelling, or chills. If you have these symptoms, stop using the medicine and get medical help right away. Tryngolza may cause high liver enzyme levels and higher levels of fat in your liver. Your health care provider may perform liver tests before you start treatment or if your dose is increased. Tell your health care provider right away if you have signs of liver problems, such as feeling tired or weak, not wanting to eat, discomfort in the upper right part of your stomach, dark urine, or yellowing of your skin and eyes.