Key takeaways:
- Olezarsen is the first FDA-approved therapy indicated to reduce acute pancreatitis risk in adults with severe hypertriglyceridemia.
- The therapy demonstrated sustained triglyceride control at 12 months.
The FDA has approved olezarsen as the first and only therapy, alongside diet, to lower triglycerides and acute pancreatitis risk in patients with severe hypertriglyceridemia, according to a manufacturer press release.
Olezarsen (Tryngolza, Ionis Pharmaceuticals), an apolipoprotein C-III inhibitor, is administered once monthly by autoinjector and is available in 50 mg and 80 mg doses. As Healio previously reported, olezarsen had been granted FDA orphan drug status in February 2024 for the treatment of familial chylomicronemia syndrome, a rare form of severe hypertriglyceridemia (sHTG).
“I have seen firsthand how challenging it can be for patients with sHTG to lower their triglycerides below 500 mg/dL, despite background lipid-lowering therapies and lifestyle changes, which leaves them at risk of a devastating and potentially life-threatening acute pancreatitis attack,” Archna Bajaj, MD, assistant professor of clinical medicine at University of Pennsylvania, said in the release. “Tryngolza is a transformational new therapy that showed unprecedented, clinically meaningful outcomes for sHTG, with the potential to redefine the treatment paradigm.”
The FDA based its approval on positive results from the phase 3 CORE and CORE2 trials, published in The New England Journal of Medicine. In the studies, olezarsen delivered significant triglyceride reductions at 6 months: The 50 mg dose lowered levels by 49% to 63% vs. placebo, while the 80 mg dose achieved reductions of 55% to 72%.
At 12 months, olezarsen was linked to significant reduction in acute pancreatitis events, with the 50 mg and 80 mg doses demonstrating 91% and 76% reductions, respectively. Additionally, 86% of patients with baseline and 12-month data achieved triglyceride levels below 500 mg/dL, a key threshold associated with lower acute pancreatitis risk.
“The approval of Tryngolza marks an historic advance for people who have long struggled to control their dangerously high triglycerides, providing the only approved therapy for sHTG to dramatically lower triglyceride levels and significantly reduce acute pancreatitis events,” Brett P. Monia, PhD, CEO at Ionis Pharmaceuticals, said in the release.
The most common adverse events for patients with sHTG included injection site reactions and liver enzyme increases.
“With limited options to lower triglycerides, people living with sHTG often face a constant and real fear that a debilitating acute pancreatitis attack could strike at any time without warning,” Emily Draud, interim executive director of the National Pancreas Foundation, said in the release. “The availability of Tryngolza for sHTG represents an important new option for this community, offering hope for people who have been waiting for a new treatment to reduce the risk of acute pancreatitis by significantly lowering their triglyceride levels.”
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