FDA Approves Tryngolza for Rare Cholesterol Disease

The FDA has approved Tryngolza (olezarsen) to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

Familial chylomicronemia syndrome is a rare, genetic disease that results in extremely high levels of triglyceride levels. Triglyceride levels in patients with this disease can be anywhere from 10 times to 100 times greater than normal.

Familial chylomicronemia syndrome is caused by impaired function of the enzyme lipoprotein lipase and, as a results, the enzyme is not able to break down chylomicrons, lipoprotein particles that are 90% triglycerides. People living with this disease are at high risk of acute pancreatitis; fatty deposits in the retina and under the skin; enlarged liver and spleen and cognitive symptoms such as brain fog, fatigue and memory loss.

Developed by Ionis Pharmaceuticals, Tryngolza is a subcutaneous RNA-targeted therapy designed to lower the body’s production of apoC-III, a protein produced in the liver that is a key regulator of triglyceride metabolism. It is the first medication approved for familial chylomicronemia syndrome. The once-monthly therapy is expected to be available by the end of the year with a price of $595,000, according to Ionis officials in an investor call.

Related: Nine Treatments for Genetic Disorders in the Offing, Says Vizient Report

The price of Tryngolza is at the high end what Vizient had estimated would the wholesale acquisition cost (WAC). In a report in June 2024, Vizient had estimated the WAC price to be set between $400,000 and $600,000.

The company is providing patients and physicians a program called Ionis Every Step that offers a suite of services, including personal support, disease and nutrition education and training, authorization assistance. Ionis is providing financial support programs including a $0 copay for commercially insured patients.

“Many people living with FCS have experienced severe pain their whole lives – sometimes so intense they require lengthy hospitalization stays – and struggle through life with daily fatigue, nausea, brain fog and stomach pain,” Lindsey Sutton Bryan, co-founder and co-president, FCS Foundation, said in a news release.

The approval was based on data from the phase 3 Balance clinical trial in adult patients with genetically identified FCS and fasting triglyceride levels ≥880 mg/dL. In the Balance study, Tryngolza 80 mg demonstrated a statistically significant placebo-adjusted mean reduction in triglyceride levels of 42.5% from baseline to six months. At 12 months Tryngolza achieved a placebo-adjusted 57% mean reduction in triglycerides.

Tryngolza also demonstrated a clinically meaningful reduction in acute pancreatitis events over 12 months, and an 84% reduction in all cause hospitalizations.

The most common adverse reactions were injection site reactions, decreased platelet count and joint pain.

Results of the Balance trial were published in April 2024 in The New England Journal of Medicine.