Familial chylomicronemia syndrome (FCS) is a rare genetic disease that causes very high triglyceride levels in the body.
Tryngolza (olezarsen) was approved in 2024 and is the first and only FDA-approved medicine to treat people with FCS along with a low-fat diet.
What Is Familial Chylomicronemia Syndrome (FCS)?
FCS is a rare genetic disease that affects how fat is broken down in your body. People who have FCS have very high levels of a type of fat known as triglycerides in the blood. A normal triglyceride level is 150 mg/dL or lower. People with FCS can have triglyceride levels of 1,000 mg/dL or higher.
High levels of triglycerides in the body can cause a life-threatening and painful condition called pancreatitis, which is an inflammation of your pancreas. It can also cause severe stomach pain, fat deposits in your skin, as well as changes to your eyes, liver, and spleen.
How Does Tryngolza Work for Familial Chylomicronemia Syndrome?
Tryngolza helps your body break down fats and lower triglyceride levels. It does this by lowering the amount of a certain protein in your body known as apolipoprotein C-III (apoC-III). ApoC-III slows down the removal of triglycerides from the body, so by reducing the amount of apoC-III, Tryngolza can help remove more triglycerides from your body.
Tryngolza is intended to be used with a low-fat diet. People taking Tryngolza should eat less than or equal to 20 grams of fat per day.
How Was Tryngolza Studied?
Tryngolza was studied in 66 adults with FCS, which was confirmed with a genetic test. The people in the study were treated monthly for about a year with either Tryngolza 80 mg, Tryngolza 50 mg, or a placebo shot. Placebo shots do not contain any active medicine.
People in this study had to have a stable diet for at least two weeks and were told to eat 20 grams or less of total fat per day while they were taking Tryngolza in the study. They were also told not to drink any alcohol during the study.
The investigators of this study looked to see if there were any improvements in fasting triglyceride levels at six and 12 months. They also looked at the number of episodes of acute pancreatitis that people had during the study.
What types of people were included in the study?
The average age for people in this study ranged from 44 to 47 years, and about half were female. People in this study were mostly White (77% to 96%), seven people were Hispanic or Latino, six were Asian, one was Native Hawaiian or Pacific Islander, and two reported a race of “other.”
Most people (65% to 77%) reported having had pancreatitis within the past 10 years. For these people, the average number of times they had pancreatitis ranged from four to six times.
At the start of the study, the average triglyceride level was about 2,500 mg/dL.
Most people were taking medications to lower cholesterol or triglycerides in the body such as.
- Statins (19%-30%)
- Omega-3-fatty acids (29%-55%)
- Fibrates (38%-50%)
What are the main benefits of Tryngolza based on the study?
After six months, people who received the 80 mg dose of Tryngolza had about a 30% decrease in their fasting triglyceride levels. And people who received the placebo had an increase in their fasting triglyceride levels by about 12%.
Your results may differ from what was seen in clinical studies.
What side effects of Tryngolza were seen in the study?
The most common side effects reported in this study are included in the table below.
| Side effect | Tryngolza 80 mg (22 people) | Placebo (23 people) |
| Coronavirus 2019 | 14% | 35% |
| Stomach pain | 18% | 35% |
| Diarrhea | 9% | 26% |
| Headache | 5% | 13% |
| Pancreatitis | 5% | 17% |
| Fatigue | 5% | 17% |
Injection site reactions were seen in 9%-15% of people in this study, and they were all graded as mild.
In this study, there were 13 episodes of pancreatitis reported.
- One episode in a patient who received the 80 mg dose
- One episode in a patient who received the 50 mg dose
- 11 episodes in seven patients who received the placebo shot
Your results may differ from what was seen in clinical studies.
How Long Until I See the Full Effects of Tryngolza?
In the clinical study, people began to see improvements in their fasting triglyceride levels right away, with the largest change occurring in the first month. These results continued through 12 months.
How Do I Manage Side Effects?
The most common side effect people have with Tryngolza is injection site reactions, which may include redness or pain in the area where you injected the medicine. To help avoid injection site reactions, make sure you are injecting the medicine in a different spot each time. This is known as rotating the injection site. Take the drug out of the refrigerator and allow it to warm to room temperature for 30 minutes. Contact your health care provider if you have questions on how to take your injection.
People treated with Tryngolza might also have a decreased platelet count and joint pain. Let your health care provider know if you have any unexplained bruises, skin discoloration, or joint pain or stiffness.
Is There Any Cost Assistance Available for Tryngolza?
There may be cost assistance to help you afford Tryngolza. You may have options even if you do not have health insurance. Ionis Every Step is a service that can help with access to medications and coordination of care. Your health care provider can help you get started, or you can call 844-789-8744.
What Else Should I Know About Tryngolza?
It is important to still follow a low-fat diet and avoid alcohol while you are taking Tryngolza. Tryngolza is not a substitute for a low-fat diet.