Exciting news! The U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA (olezarsen), developed by Ionis, as the first-ever treatment for Familial Chylomicronemia Syndrome (FCS). FCS is a rare genetic condition that causes dangerously high triglyceride levels, increasing the risk of severe health complications–especially pancreatitis. This approval is a major milestone—not just for people with FCS, but for the entire pancreatitis community.
What is Familial Chylomicronemia Syndrome (FCS)?
FCS is a rare genetic disorder that prevents the body from properly breaking down fats in the blood, leading to dangerously high triglyceride levels. Triglycerides are a type of fat that the body uses for energy, but in people with FCS, levels can be more than five times higher than normal.
People with FCS often experience:
- Severe stomach pain
- Numbness in feet or legs
- Vomiting or diarrhea
- Forgetfulness and fatigue
- Frequent hospitalizations
- Pancreatitis
FCS Foundation
The FCS Foundation has resources including information about genetic testing for Familial Chylomicronemia Syndrome (FCS) patients, caregivers and family members.
More severe symptoms can include lipemia retinalis, neurological symptoms, hepatosplenomegaly, xanthomas, and life-threatening attacks of acute pancreatitis.
Although FCS affects about 3,000 people in the U.S., it has a serious impact on those who live with it. This condition is also known as lipoprotein lipase deficiency (LPLD) or familial hypertriglyceridemia and is a rare genetic form of severe hypertriglyceridemia (s-HTG).
Read more about Familial Chylomicronemia Syndrome (FCS)
How Does This Treatment Work?
This newly approved treatment lowers APOC3, a protein that plays a key role in regulating fat (triglyceride) levels in the blood. By reducing APOC3, the drug helps decrease triglycerides and lower the risk of pancreatitis attacks in people with FCS.
Eligibility
Currently, the FDA has approved TRYNGOLZA for patients with Familial Chylomicronemia Syndrome in combination with a low fat diet.
Why Does This Matter for the Pancreatitis Community?
Even though FCS is rare, this approval is a big deal for all people with pancreatitis. Here’s why:
- It opens the door for new pancreatitis treatments. Before this approval, there was no FDA-approved drug to reduce pancreatitis attacks. Having a treatment for FCS shows that targeting triglycerides can help prevent pancreatitis, which could encourage more research into treatments for other causes of the disease.
- It makes genetic testing more useful. Many people with pancreatitis never find out what’s causing their condition because doctors hesitate to order genetic tests when there’s no treatment available. But now that there’s a treatment for a genetic cause of pancreatitis, doctors may be more likely to recommend genetic testing to help guide care.
- It shows biotech companies and investors that pancreatitis research is worth funding. Drug companies and investors are often hesitant to develop new pancreatitis treatments because there hasn’t been a clear way to get them FDA-approved. This approval—and others that may follow—proves that treatments can move from research to reality.
For more details, you can read the official press release here.
Looking Ahead
This isn’t the only exciting news in the fight against high triglycerides and pancreatitis. Arrowhead Pharmaceuticals recently submitted a New Drug Application (NDA) to the FDA for Plozasiran, another potential treatment designed to lower triglycerides by targeting the APOC3 protein. If approved, it could offer another option for people with severe hypertriglyceridemia and FCS. The progress of multiple drugs in this space signals growing momentum in pancreatitis research, bringing hope for more treatment options in the future.
This FDA approval is an important step forward. While it currently applies to only a small portion of the pancreatitis community, it paves the way for further research and new treatment options. At Mission: Cure, we will continue working toward more effective treatments for all forms of pancreatitis—because everyone deserves a path to relief.
About Mission: Cure
At Mission: Cure, we are dedicated to finding effective treatments and, ultimately, a cure for pancreatitis. Through patient education, research, and advocacy, we strive to improve the lives of those affected by this condition.
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