Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) has officially launched Tryngolza™ (olezarsen), a new RNA-targeted therapy for Familial Chylomicronemia Syndrome (FCS). Approved by the U.S. FDA in December 2024, Tryngolza™ is the first FDA-approved treatment for FCS, a rare and life-threatening genetic disorder. The annual list price for Tryngolza™ is $595,000.
What is Familial Chylomicronemia Syndrome (FCS)?
FCS is an ultra-rare condition that affects around 1 to 2 people per million worldwide. It causes extremely high triglyceride levels in the blood and leads to recurrent acute pancreatitis, a serious and sometimes fatal complication. FCS is caused by mutations in genes responsible for lipid metabolism. Patients typically face strict dietary restrictions, which have limited effectiveness in controlling the disease.
Traditional treatments have not been sufficient. Tryngolza™ offers a novel approach to treating FCS by targeting the root causes of the condition.
Phase 3 Study Results: Positive Impact on Triglycerides
The approval of Tryngolza™ was based on the results of the Phase 3 BALANCE trial, which showed significant improvements in patients’ triglyceride levels. The study found:
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A 44% reduction in triglycerides after 6 months of treatment (placebo-adjusted).
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A 59% reduction after 12 months (p<0.001).
In addition to reducing triglycerides, Tryngolza™ also significantly lowered apoC-III levels. ApoC-III is a protein involved in triglyceride regulation. Lowering apoC-III may reduce the risk of heart disease and pancreatitis. The therapy also helped decrease the frequency of acute pancreatitis episodes, offering significant clinical benefits for FCS patients.
Convenient Once-Monthly Dosing and Flexible Storage
Tryngolza™ is administered through a subcutaneous injection using a prefilled 80 mg/0.8 mL autoinjector, once every 4 weeks. This once-monthly dosing is designed for patient convenience. The therapy can be stored in a refrigerator (2–8°C) or at room temperature for up to six weeks, offering flexibility in storage and use.
Pricing and Access to Treatment
The annual price for Tryngolza™ is $595,000. Ionis Pharmaceuticals is committed to ensuring that financial barriers do not prevent patients from accessing this therapy. The company will work closely with insurance providers to ensure broad coverage.
In addition, Ionis is launching patient support programs to help with financial assistance and co-pay support. These programs are designed to ease the burden for patients and improve their access to Tryngolza™.
Ionis has also partnered with DengYueMed, a drug distributor in Hong Kong, to improve the affordability of Tryngolza™ in the region. DengYueMed provides transparent pricing and reliable support to ensure that patients can access the therapy without delays.
CEO’s Statement
Brett P. Monia, Ph.D., CEO of Ionis Pharmaceuticals, said: “Today marks a historic milestone for the FCS community. Tryngolza™ offers a critical treatment option that can effectively lower triglyceride levels and reduce the risk of dangerous complications. We are proud to bring this therapy to patients in the U.S. and look forward to expanding access globally.”
Plans for Global Expansion
Following the U.S. launch, Ionis Pharmaceuticals plans to seek regulatory approval for Tryngolza™ in Europe and other international markets by the end of 2025. This will bring the benefits of Tryngolza™ to more patients around the world.
About Ionis Pharmaceuticals
Ionis Pharmaceuticals is a leader in RNA-targeted therapies. The company focuses on developing treatments for rare and severe diseases. Ionis is committed to transforming the treatment landscape for patients with high unmet medical needs. Tryngolza™ adds to Ionis’ growing portfolio of innovative therapies.
About DengYueMed
DengYueMed is a certified drug distributor based in Hong Kong. The company is dedicated to providing affordable access to medicines for patients in the region. DengYueMed is fully certified by the Pharmacy and Poisons Board of Hong Kong and works to ensure that all patients can access Tryngolza™ in a timely and cost-effective manner.
For more information on Tryngolza™ or its availability in Hong Kong, please contact DengYueMed directly.
