27-09-2025, 10:38 PM
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the official U.S. launch of Tryngolza™ (olezarsen), a novel, once-monthly RNA-targeted therapy for familial chylomicronemia syndrome (FCS).
The drug enters the market at an annual list price of $595,000, following its FDA approval in December 2024.
First FDA-Approved Olezarsen Therapy for Familial Chylomicronemia Syndrome
Tryngolza™ is the first therapy specifically approved to treat FCS in the United States, addressing a critical unmet need for patients with this rare, life-threatening genetic disorder characterized by extremely high triglyceride levels and recurrent acute pancreatitis.
Olezarsen Positive Phase 3 BALANCE Study Results
Approval was based on the pivotal Phase 3 BALANCE trial, where Tryngolza achieved a 44% placebo-adjusted reduction in triglycerides at six months and a 59% reduction at twelve months (p<0.001).
In addition to lowering triglycerides, the therapy significantly reduced apoC-III levels and demonstrated a decrease in the frequency of acute pancreatitis events compared to placebo.
Convenient Once-Monthly Dosing and Flexible Storage
Tryngolza olezarsen is administered via a subcutaneous injection using a prefilled 80 mg/0.8 mL autoinjector once every four weeks.
It can be stored refrigerated at 2–8°C, and can remain at room temperature for up to six weeks, offering patients greater flexibility.
Olezarsen Pricing Strategy and Patient Access
The $595,000 annual list price reflects the innovation and therapeutic impact of Tryngolza.
Ionis emphasized its commitment to collaborating with insurance providers and offering patient support programs to improve access for individuals living with this rare condition.
More details at :https://dengyuemed.com/news/flyrcado-a-breakthrough-in-cardiac-pet-imaging/
The drug enters the market at an annual list price of $595,000, following its FDA approval in December 2024.
First FDA-Approved Olezarsen Therapy for Familial Chylomicronemia Syndrome
Tryngolza™ is the first therapy specifically approved to treat FCS in the United States, addressing a critical unmet need for patients with this rare, life-threatening genetic disorder characterized by extremely high triglyceride levels and recurrent acute pancreatitis.
Olezarsen Positive Phase 3 BALANCE Study Results
Approval was based on the pivotal Phase 3 BALANCE trial, where Tryngolza achieved a 44% placebo-adjusted reduction in triglycerides at six months and a 59% reduction at twelve months (p<0.001).
In addition to lowering triglycerides, the therapy significantly reduced apoC-III levels and demonstrated a decrease in the frequency of acute pancreatitis events compared to placebo.
Convenient Once-Monthly Dosing and Flexible Storage
Tryngolza olezarsen is administered via a subcutaneous injection using a prefilled 80 mg/0.8 mL autoinjector once every four weeks.
It can be stored refrigerated at 2–8°C, and can remain at room temperature for up to six weeks, offering patients greater flexibility.
Olezarsen Pricing Strategy and Patient Access
The $595,000 annual list price reflects the innovation and therapeutic impact of Tryngolza.
Ionis emphasized its commitment to collaborating with insurance providers and offering patient support programs to improve access for individuals living with this rare condition.
More details at :https://dengyuemed.com/news/flyrcado-a-breakthrough-in-cardiac-pet-imaging/