Ionis Pharmaceuticals - Tryngolza to be available in U.S. for SHTG in July

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has made significant progress in its regulatory and commercial development of TRYNGOLZA® (olezarsen), with the U.S. Food and Drug Administration (FDA) accepting its supplemental New Drug Application (sNDA) for the treatment of severe hypertriglyceridemia (sHTG) under Priority Review. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026, suggesting potential approval by that date. If approved, TRYNGOLZA would become the first FDA-approved treatment for sHTG, a condition affecting approximately 3 million individuals in the U.S., with over 1 million considered high risk.

The sNDA submission was supported by robust data from the Phase 3 CORE and CORE2 trials, which demonstrated statistically significant reduction in triglycerides and acute pancreatitis events in patients treated with olezarsen. These findings led to a revision of the drug’s peak net sales guidance for sHTG, with expectations now exceeding $2 billion annually. This represents a substantial increase from the previous guidance of over $1 billion, reflecting the drug’s strong clinical profile and market potential.

Ionis has also highlighted the broader momentum in its pipeline, with multiple late-stage programs expected across both owned and partnered assets expected to deliver key regulatory and clinical milestones in 2026. The company aims to achieve cash flow breakeven by 2028, driven by continued revenue growth from its commercial products and anticipated launches. With the FDA’s Priority Review designation and the strong performance of TRYNGOLZA in its initial indication for familial chylomicronemia syndrome (FCS), Ionis is positioned to expand its market presence in the cardiometabolic disease space.