Ionis Pharmaceuticals is dramatically lowering the price of its blockbuster hopeful Tryngolza (olezarsen) as it prepares to grow the med’s reach with a planned label expansion.
Initially approved as a treatment for familial chylomicronemia syndrome (FCS) in 2024, the drug first launched with an annual list price of $595,000, the company has said. Now, as Ionis gears up to potentially launch in severe hypertriglyceridemia (sHTG), the company is rolling out an updated wholesale acquisition cost (WAC) of $40,000 annually, it announced Wednesday.
The new price point “reflects the substantial value that olezarsen can provide to patients, healthcare professionals and the healthcare system, while supporting timely and sustained patient access,” the company said, adding that the decision follows a “thorough review of robust provider research and extensive payer engagement.”
Tryngolza’s new price will take effect April 1 and will apply to both the anticipated sHTG indication and its current use in FCS. With the FDA expected to decide on its sHTG application by June 30, changing the price ahead of time allows for “proactive alignment” with annual payer contracting cycles while ensuring a “seamless launch” and speedy access post approval, the company said.
Payer contracting cycles are expected to begin in April, allowing Ionis to get in on the talks and reap the “first-mover advantage” in the sizable market, Leerink analysts explained in a note to clients, effectively positioning the drug for strong uptake in 2027.
Not only does the sharp WAC decrease support broader access and speak to the higher cost of drugs in the rare disease realm, but it could also give Ionis a better shot against its market rival.
The $40,000 price point offers Ionis a pricing edge on Arrowhead Pharmaceuticals and its competing Redemplo, which hit the FCS scene in November. Redemplo lists for $60,000 annually before any negotiations, putting Ionis in a “sweet spot” to contract “aggressively,” the Leerink analysts pointed out.
Ionis’ Tryngolza has been underpinned by substantial hype leading up to its potential sHTG launch, following the unveiling of what William Blair analyst Myles Minter, Ph.D., called “best-case scenario” data from the largest sHTG study ever run.
In a pair of phase 3 trials, the drug demonstrated placebo-adjusted reductions of 55% and 72% in triglycerides after six months of treatment. In addition, treated patients in the studies saw an 85% lower risk of acute pancreatitis over a year of treatment.
The results exceeded the William Blair team’s expectations, Minter wrote at the time, while Citi analysts called the findings a “home run.” The FDA opted to take Ionis’ expansion bid under its priority review track in February, which CEO Brett Monia, Ph.D., said was a “significant step toward our goal of delivering the first-ever treatment shown to reduce the risk of potentially life-threatening acute pancreatitis attacks in people with sHTG.”
“We believe that this is an unprecedented and historical achievement in the field of lipidology, where the quest to reduce the risk of acute pancreatitis in sHTG has been ongoing for nearly 50 years,” Sam Tsimikas, M.D., Ionis’ chief of global cardiovascular development, added on a webcast after the trial results dropped in September.
Severe hypertriglyceridemia is hallmarked by very high triglycerides and an increased risk of acute pancreatitis and hospitalization. With Tryngolza, which is designed to lower the body’s production of triglyceride metabolism-regulating protein apoC-III, Ionis is targeting a U.S. patient population of 3 million people, including 1 million sHTG patients who are considered high risk.
Eying a largely untapped commercial treatment market in sHTG, Ionis figures Tryngolza could reach peak sales of $2 billion-plus across both of its combined indications. The company doubled its peak revenue forecast for the drug in January based on research that suggested a significant increase in projected sales volumes compared to the company’s previous expectation, Monia said in a presentation at the time.
Tyrngolza in FCS was Ionis’ first solo product launch after years of licensing out its drugs to development partners. The drug generated $108 million in 2025 sales after its FDA nod the prior year.
Arrowhead remains a few steps behind Ionis on the regulatory trail, earning a breakthrough therapy designation for its Redemplo in sHTG at the end of last year. Arrowhead’s FCS nod came a year after Tryngolza crossed the FDA finish line in the same indication and followed a bitter legal dispute in which Ionis accused Arrowhead of copying its mRNA technology to make Redemplo.
