RARE Daily

FDA Grants Fast Track Designation for Ionis’ Olezarsen in Patients with Familial Chylomicronemia Syndrome

February 1, 2023

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation for Ionis Pharmaceuticals’ olezarsen for the treatment of familial chylomicronemia syndrome.

Familial chylomicronemia syndrome (FCS) is a rare, genetic disease that is characterized by extremely elevated triglyceride levels. FCS can lead to many chronic health issues including severe, recurrent abdominal pain, fatigue, high risk of life-threatening pancreatitis and abnormal enlargement of the liver or spleen. In addition, people with FCS are often unable to work, adding to their disease burden. In severe cases, patients can have bleeding into the pancreas, serious tissue damage, infection, and cyst formation, as well as damage to other vital organs such as the heart, lungs, and kidneys. There are currently no approved therapies for the treatment of FCS in the United States.

Olezarsen  is an investigational LICA medicine designed to inhibit the production of apoC-III for patients who are at risk of disease due to elevated triglyceride levels. ApoC-III is a protein produced in the liver that regulates triglyceride metabolism in the blood. People with severely elevated triglycerides, such as people with FCS, are at high risk for acute pancreatitis and an increased risk of cardiovascular disease.

Ionis fully enrolled its global phase 3 BALANCE study of olezarsen in adult patients with FCS last year. The company plans to share data from the BALANCE study in the second half of 2023. In addition to FCS, Ionis is evaluating olezarsen in severe hypertriglyceridemia (SHTG).

Fast Track designation is designed to expedite the FDA’s review of innovative, new drugs that demonstrate the potential to address unmet medical need.

“The FDA Fast Track designation for olezarsen recognizes the urgent need for an effective treatment for FCS, a debilitating rare disease affecting people with very limited treatment options and an elevated risk of painful and potentially fatal bouts of pancreatitis,” said Richard Geary, executive vice president and chief development officer at Ionis.

Photo: Richard Geary, executive vice president and chief development officer at Ionis.

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