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.

Executive Statement from Ionis Pharmaceuticals

"Today marks a historic milestone for the FCS community," said Brett P. Monia, Ph.D., Chief Executive Officer of Ionis. "

Tryngolza provides a much-needed option that can effectively lower triglyceride levels and reduce the risk of serious, potentially fatal complications."

Addressing a Rare, High-Burden Disease

FCS affects approximately 1 to 2 people per million, and managing the disease has traditionally relied on strict dietary fat restrictions with limited success.

Tryngolza offers new hope for patients suffering from this devastating condition.

Future Plans for Global Expansion

Following the U.S. launch, Ionis plans to seek regulatory approval in Europe and other global markets by the end of 2025, expanding access to patients worldwide.

About Ionis Pharmaceuticals

Ionis Pharmaceuticals is a pioneer in RNA-targeted therapies, dedicated to transforming the lives of people with severe and rare diseases.

Tryngolza adds to Ionis’ expanding portfolio of commercialized therapies aimed at high unmet needs.

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