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FDA Expands IONS' Tryngolza Label to Treat Severe Hypertriglyceridemia
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Key Takeaways
Ionis won FDA approval to expand Tryngolza to treat severe hypertriglyceridemia in adults.
IONS plans a launch next month after phase III studies met primary endpoints and cut triglycerides.
Tryngolza generated $108 million in 2025 sales and $27 million in first-quarter 2026 revenue.
Ionis Pharmaceuticals (IONS - Free Report) announced that the FDA has approved its wholly owned drug, Tryngolza (olezarsen), for a new indication. The drug has been cleared as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis (AP) in adults with severe hypertriglyceridemia (sHTG).
The FDA’s nod makes Tryngolza the first approved treatment for sHTG in the United States. Ionis plans to commercially launch the drug for this new indication next month.
The approval is supported by data from two phase III studies — CORE and CORE2 — both of which met their primary endpoints. Treatment with the drug showed statistically significant reductions in triglyceride (TG) levels across both 80 mg and 50 mg monthly doses. Both studies also showed a significant reduction in AP events, a key secondary endpoint. The approval comes ahead of the June 30 target action date previously set by the FDA.
TG is a type of fat in the blood that, at elevated levels, increases the risk of heart disease and pancreatitis. People with sHTG, marked by TG levels above 500 mg/dL, are at higher risk of potentially life-threatening acute pancreatitis (AP), as well as atherosclerotic cardiovascular disease (ASCVD).
Year to date, shares of Ionis have lost over 3% compared with the industry’s nearly 6% fall.
Image Source: Zacks Investment Research
Tryngolza — A Key Growth Driver for Ionis
The latest FDA approval significantly expands Tryngolza's commercial opportunity. Per Ionis, nearly 3 million people in the United States are living with sHTG. As the first FDA-approved treatment for the condition, the drug enjoys a first-mover advantage, giving the company access to a significantly broader patient population and a potential blockbuster market.
Even before securing this expanded approval, Tryngolza had emerged as a key growth driver for Ionis. Despite initially being approved for the much smaller FCS indication, the drug's commercial launch has been encouraging. Ionis generated $108 million in Tryngolza product sales in 2025, followed by another $27 million in the first quarter of 2026.
Tryngolza was also approved in the European Union in October 2025 for FCS. In March 2025, Ionis out-licensed the drug's ex-U.S. rights to Sweden-based Sobi. Under the agreement, Sobi will handle all future regulatory filings and commercialization activities outside the United States, excluding Canada and China. A regulatory filing is currently under review by the EMA, seeking to expand Tryngolza's label to include sHTG.
Ionis Boasts a Diverse Revenue Stream
Ionis has collaborations with leading drugmakers/biotech companies, such as AstraZeneca (AZN - Free Report) , Biogen (BIIB - Free Report) , GSKplc (GSK - Free Report) and Novartis. These deals provide the company with funds in the form of license fees, upfront and milestone payments to invest in the development of its wholly owned pipeline.
Ionis earns commercial revenues in the form of royalty payments on net sales of Spinraza, approved in the United States to treat spinal muscular atrophy (SMA) worldwide. The company licensed this drug to Biogen, which is responsible for its commercialization. Ionis and Biogen also market Qalsody, which was approved by the FDA in 2023 for amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 mutations.
AstraZeneca, Novartis and GSK are its partners for Wainua, pelacarsen and bepirovirsen, respectively. The FDA approved Wainua in 2023 to treat adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (ATTRv-PN) in the United States. The drug is also being developed in a late-stage study to treat transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), an indication with a larger market than ATTRv-PN. Data from this study is expected later this year.
GSK-partnered bepirovirsen is currently under FDA review for chronic hepatitis B, with a regulatory decision expected by Oct. 26, 2026. Novartis-partnered pelacarsen is also in late-stage development for elevated lipoprotein(a)-driven cardiovascular disease, with a readout expected before this year’s end.
Supported by these partnership revenues, Ionis continues to expand its wholly owned commercial portfolio. Following the approvals of Tryngolza and Dawnzera (for HAE), the company expects additional product launches over the next one to two years. These include zilganersen for Alexander disease, which is currently under FDA review with a Sept. 22, 2026 PDUFA date, and obudanersen for Angelman syndrome, which is being evaluated in a phase III study, with data expected in 2027.
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FDA Expands IONS' Tryngolza Label to Treat Severe Hypertriglyceridemia
Key Takeaways
Ionis Pharmaceuticals (IONS - Free Report) announced that the FDA has approved its wholly owned drug, Tryngolza (olezarsen), for a new indication. The drug has been cleared as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis (AP) in adults with severe hypertriglyceridemia (sHTG).
The FDA’s nod makes Tryngolza the first approved treatment for sHTG in the United States. Ionis plans to commercially launch the drug for this new indication next month.
The approval is supported by data from two phase III studies — CORE and CORE2 — both of which met their primary endpoints. Treatment with the drug showed statistically significant reductions in triglyceride (TG) levels across both 80 mg and 50 mg monthly doses. Both studies also showed a significant reduction in AP events, a key secondary endpoint. The approval comes ahead of the June 30 target action date previously set by the FDA.
TG is a type of fat in the blood that, at elevated levels, increases the risk of heart disease and pancreatitis. People with sHTG, marked by TG levels above 500 mg/dL, are at higher risk of potentially life-threatening acute pancreatitis (AP), as well as atherosclerotic cardiovascular disease (ASCVD).
Tryngolza was approved by the FDA in 2024 to treat familial chylomicronemia syndrome (FCS), a rare form of sHTG marked by extremely elevated TG levels (>880 mg/dL). Following that approval, Tryngolza became the first FDA-approved treatment for FCS and marked Ionis' first commercial product launch.
IONS Stock Performance
Year to date, shares of Ionis have lost over 3% compared with the industry’s nearly 6% fall.
Image Source: Zacks Investment Research
Tryngolza — A Key Growth Driver for Ionis
The latest FDA approval significantly expands Tryngolza's commercial opportunity. Per Ionis, nearly 3 million people in the United States are living with sHTG. As the first FDA-approved treatment for the condition, the drug enjoys a first-mover advantage, giving the company access to a significantly broader patient population and a potential blockbuster market.
Even before securing this expanded approval, Tryngolza had emerged as a key growth driver for Ionis. Despite initially being approved for the much smaller FCS indication, the drug's commercial launch has been encouraging. Ionis generated $108 million in Tryngolza product sales in 2025, followed by another $27 million in the first quarter of 2026.
Tryngolza was also approved in the European Union in October 2025 for FCS. In March 2025, Ionis out-licensed the drug's ex-U.S. rights to Sweden-based Sobi. Under the agreement, Sobi will handle all future regulatory filings and commercialization activities outside the United States, excluding Canada and China. A regulatory filing is currently under review by the EMA, seeking to expand Tryngolza's label to include sHTG.
Ionis Boasts a Diverse Revenue Stream
Ionis has collaborations with leading drugmakers/biotech companies, such as AstraZeneca (AZN - Free Report) , Biogen (BIIB - Free Report) , GSK plc (GSK - Free Report) and Novartis. These deals provide the company with funds in the form of license fees, upfront and milestone payments to invest in the development of its wholly owned pipeline.
Ionis earns commercial revenues in the form of royalty payments on net sales of Spinraza, approved in the United States to treat spinal muscular atrophy (SMA) worldwide. The company licensed this drug to Biogen, which is responsible for its commercialization. Ionis and Biogen also market Qalsody, which was approved by the FDA in 2023 for amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 mutations.
AstraZeneca, Novartis and GSK are its partners for Wainua, pelacarsen and bepirovirsen, respectively. The FDA approved Wainua in 2023 to treat adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (ATTRv-PN) in the United States. The drug is also being developed in a late-stage study to treat transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), an indication with a larger market than ATTRv-PN. Data from this study is expected later this year.
GSK-partnered bepirovirsen is currently under FDA review for chronic hepatitis B, with a regulatory decision expected by Oct. 26, 2026. Novartis-partnered pelacarsen is also in late-stage development for elevated lipoprotein(a)-driven cardiovascular disease, with a readout expected before this year’s end.
Supported by these partnership revenues, Ionis continues to expand its wholly owned commercial portfolio. Following the approvals of Tryngolza and Dawnzera (for HAE), the company expects additional product launches over the next one to two years. These include zilganersen for Alexander disease, which is currently under FDA review with a Sept. 22, 2026 PDUFA date, and obudanersen for Angelman syndrome, which is being evaluated in a phase III study, with data expected in 2027.
Ionis Pharmaceuticals, Inc. Price
Ionis Pharmaceuticals, Inc. price | Ionis Pharmaceuticals, Inc. Quote
IONS’ Zacks Rank
Ionis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.