Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) revealed today that it has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for its experimental drug olezarsen. This designation is specifically for the treatment of familial chylomicronemia syndrome (FCS), a rare genetic disorder marked by significantly elevated triglyceride levels and recurrent acute pancreatitis (AP). Orphan Drug designation is granted by the FDA to medications aimed at treating, preventing, or diagnosing rare diseases or conditions that affect fewer than 200,000 individuals in the United States at the time of designation.
“People afflicted with FCS are in critical need of a medication that could potentially stabilize their triglyceride levels and mitigate the peril of life-threatening AP episodes,” stated Sam Tsimikas, M.D., senior vice president of global cardiovascular development at Ionis. “Should olezarsen receive approval, it holds promise to become the standard treatment for FCS in the United States. We are fully dedicated to advancing this significant medication, which carries the potential to bring about transformative changes for individuals and families grappling with FCS.”
FCS obstructs the body’s ability to metabolize fats, resulting in fasting triglyceride levels that can surge to levels ranging from 10 to 100 times higher than normal. Individuals with FCS face a heightened risk of AP, characterized by inflammation of the pancreas, often leading to hospitalization and posing life-threatening circumstances. In severe instances, vital organs such as the heart, lungs, kidneys, and pancreas can sustain damage.
Through the FDA’s Orphan Drug Act, orphan drug designation offers incentives such as tax credits, grants, waivers for certain clinical trial administrative fees, and seven years of market exclusivity post-drug approval.
