FDA Approves Ionis Pharmaceuticals’ Tryngolza for Familial Chylomicronemia Syndrome

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The FDA has approved Ionis Pharmaceuticals’ Tryngolza (olezarsen), the first-ever treatment for adults with familial chylomicronemia syndrome (FCS) that that significantly and substantially reduces triglyceride levels and provides clinically meaningful reduction in acute pancreatitis events when used with an appropriate diet. According to the company, the approval was based on results from the Phase III Balance study, which demonstrated a clinically meaningful placebo-adjusted triglyceride reduction after 12 months of treatment.¹

"Today's FDA approval of Tryngolza heralds the arrival of the first-ever FCS treatment in the U.S. – a transformational moment for patients and their families. For the first time, adults with FCS can now access a treatment that substantially reduces triglycerides and the risk of debilitating and potentially life-threatening acute pancreatitis," said Brett P. Monia, PhD, CEO, Ionis, in a press release. "We are proud of our long-standing partnership with the FCS community and are grateful to the patients, families and investigators who participated in our clinical studies, enabling Ionis to make this new treatment a reality. The FDA approval of Tryngolza is also a pivotal moment for Ionis, representing our evolution into a fully integrated commercial-stage biotechnology company – a goal we set out to achieve five years ago. With our rich pipeline of potentially life-changing medicines, we expect Tryngolza to be the first in a steady cadence of innovative medicines we will deliver independently to people living with serious diseases."

The Balance study was a global, multicenter, randomized, double-blind, placebo-controlled trial that enrolled 66 patients over 18 years of age with confirmed FCS. As part of the study, patients received background therapies including statins, fibrates and omega-3 fatty acids. Additionally, patients were randomized to receive Tryngolza 80 mg or 50 mg or placebo via subcutaneous injection once every four weeks for 53 weeks. The primary endpoint of the study was the percent change from baseline in fasting triglyceride levels at six months compared to placebo, while secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters, and adjudicated acute pancreatitis event rates over the treatment period.²

Results found that Tryngolza demonstrated a placebo-adjusted triglyceride reduction of 42.5% at six months and 57% at 12 months, along with a substantial reduction in AP episodes.¹ Additionally, patients treated with Tryngolza experienced an 84% reduction in all-cause hospitalizations between baseline and 12 months and considerably fewer AP events during the 12-month period, compared to placebo.²

The drug exhibited a favorable safety profile, with injection site reactions, decreased platelet count, and arthralgia as the most common adverse events. Currently, it is being evaluated in three Phase III clinical trials for the treatment of severe hypertriglyceridemia.¹

"With no treatment options previously available, we were limited to relying only on extremely strict diet and lifestyle changes as the sole preventative treatment option," said Alan Brown, MD, FNLA, FACC, FAHA, clinical professor of medicine, Rosalind Franklin University of Medicine and Science; Balance trial investigator, in the press release. "The FDA approval of Tryngolza is an important moment for people living with FCS, their families and physicians who now, for the first time, have a treatment that significantly lowers triglycerides and decreases the risk of potentially life-threatening acute pancreatitis events, as an adjunct to a low-fat diet. I am excited to have a medicine I can prescribe to my patients that has been shown to change the course of their disease."

References

1. TRYNGOLZA™ (olezarsen) approved in U.S. as first-ever treatment for adults living with familial chylomicronemia syndrome as an adjunct to diet. Ionis. December 20, 2024. Accessed December 23, 2024. https://ir.ionis.com/news-releases/news-release-details/tryngolzatm-olezarsen-approved-us-first-ever-treatment-adults

2. Ionis presents positive results from Phase 3 Balance study of olezarsen for familial chylomicronemia syndrome. Ionis. April 7, 2024. Accessed December 23, 2024. https://ir.ionis.com/news-releases/news-release-details/ionis-presents-positive-results-phase-3-balance-study-olezarsen