The US Food and Drug Administration has approved Ionis Pharmaceuticals’ Tryngolza (olezarsen) for the treatment of severe hypertriglyceridemia – a dangerous condition in which triglyceride levels are critically elevated. Currently, there are no drugs on the market specifically developed for this serious condition or to reduce associated pancreatitis attacks.
Standard therapy includes generics and fish oil, which provide only modest triglyceride reductions of 10‑20%, according to Ionis CEO Brett Monia. In clinical trials, Tryngolza demonstrated significantly higher efficacy: with a once‑monthly 80 mg dose, triglyceride levels fell by 72% compared with placebo at six months, while the 50 mg dose achieved a 63% reduction.
Tryngolza is an antisense oligonucleotide. In 2024, the FDA approved it for adults with familial chylomicronemia syndrome, a rare genetic disorder causing extremely high triglycerides and pancreatitis risk. Sales for that indication reached $108 million in 2025.
The latest regulatory decision opens a much larger patient pool for Ionis. Around 3.6 million people in the United States have severe hypertriglyceridemia. The company plans to focus on those at elevated risk of acute pancreatitis – approximately 500,000 patients. In April, amid growing confidence in the drug’s prospects for the new indication, Ionis raised its peak annual sales forecast for Tryngolza from $2 billion to $3 billion.
Ahead of the expanded approval, Ionis’s commercial team had been promoting the drug to endocrinologists, cardiologists and lipid specialists since February. To improve patient access, the company took an unusual step in early April, cutting the annual cost from $595,000 to $40,000 – a move aimed at ensuring sustainable and timely supply for patients.
