Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the indication extension for TRYNGOLZA® (olezarsen) as an adjunct to diet, in combination with other lipid-lowering therapies, for the treatment of adult patients with severe hypertriglyceridemia (sHTG, fasting triglyceride levels of ≥880 mg/dL [≥10 mmol/L]). If confirmed by the European Commission (EC), TRYNGOLZA would be the first and only RNA-targeted therapy authorized in the EU for adults with sHTG and triglyceride levels ≥880 mg/dL (≥10 mmol/L). The positive CHMP opinion comes ahead of the previously anticipated timeline, with a final EC decision expected in the fourth quarter of 2026.
"Following TRYNGOLZA’s approval for sHTG in the U.S. in June, today’s positive CHMP opinion marks an important step towards making this treatment available to patients with sHTG in the EU," said Brett P. Monia, Ph.D., chief executive officer, Ionis. "The recommendation builds on the robust clinical evidence from the CORE and CORE2 studies and reflects the continued collaboration between Ionis and Sobi as we work towards a potential European approval."
The positive CHMP opinion is based on results from the pivotal Phase 3 CORE and CORE2 studies. These studies met their primary endpoint, with TRYNGOLZA demonstrating a statistically significant placebo-adjusted mean reduction in fasting triglyceride levels of up to 72% at six months, sustained through 12 months. In a pooled analysis across the two studies, TRYNGOLZA demonstrated a statistically significant 85% reduction in adjudicated acute pancreatitis events over 12 months compared with placebo. TRYNGOLZA demonstrated a favorable safety and tolerability profile. The data were published in The New England Journal of Medicine.
sHTG is commonly defined by very high triglycerides ≥500 mg/dL (≥5.65 mmol/L) and characterized by an increased risk of acute pancreatitis and other serious health complications. Approximately two million people are living with sHTG in the EU5, including approximately 700,000 with triglyceride levels ≥880 mg/dL (≥10 mmol/L).
"The CHMP's positive opinion reflects the strength of the clinical evidence and brings us one step closer to offering TRYNGOLZA as a new treatment option to help reduce the risk of acute pancreatitis in patients with sHTG in the EU," said Lydia Abad-Franch, M.D., head of research and development and medical affairs and chief medical officer, Sobi. "Acute pancreatitis is a potentially life-threatening complication of sHTG that can place a significant burden on patients through frequent hospitalizations, intensive care treatment, irreversible complications and reduced quality of life. We look forward to the final decision from the EC."