The U.S. Food and Drug Administration has approved Ionis Pharmaceuticals’ antisense oligonucleotide Tryngolza for the management of blood lipid levels and pancreatitis risk in patients with severe hypertriglyceridemia, according to a June 25 announcement. Ionis expects to make the product available for this indication in July.
Analysts at William Blair said Wednesday that the approval represents a “major market expansion for [a] wholly owned commercial opportunity.” The firm said that severe hypertriglyceridemia “should provide substantial growth” to Tryngolza, adding that the drug will be “a likely meaningful contributor to [Ionis’] cash flow breakeven guidance in 2028.”
Ionis set its cash flow goal during the 44th J.P. Morgan Healthcare Conference in January, where CEO Brett Monia said the company is “well-positioned to deliver accelerating revenue growth.”
Tryngolza is an RNA-targeting medicine designed to reduce serum apolipoprotein C-III, which helps clear triglycerides and LDL cholesterol from the bloodstream. The drug was initially approved in 2024 for familial chylomicronemia syndrome, a rare genetic disease associated with acute pancreatitis and severe hypertriglyceridemia.
Phase 3 CORE and CORE 2 studies showed that Tryngolza reduced fasting triglyceride concentrations by up to 72% at six months compared with placebo. These reductions were sustained at twelve months. The company also reported that Tryngolza lowered acute pancreatitis events by up to 91%, making it unique among therapies for severe hypertriglyceridemia.
The updated label for Tryngolza includes information about potential increases in liver enzyme levels and hepatic fat, recommending liver enzyme testing before starting or increasing dosage. Leerink Partners wrote Thursday that they do not expect these safety considerations will hinder uptake, but anticipate physicians may start patients on a lower dose before adjusting as needed.
