FDA Approves Olezarsen for Rare Disorder

The FDA has approved olezarsen, branded as Tryngolza, as the first-ever treatment for adults with familial chylomicronemia syndrome (FCS), a rare genetic disorder that leads to dangerously high triglyceride levels and acute pancreatitis. This groundbreaking drug, an antisense oligonucleotide, is indicated as an adjunct to a low-fat diet and demonstrated a significant reduction in triglyceride levels by 42.5% in clinical trials. The approval marks a transformative moment for patients, as there were previously no effective treatment options, relying solely on strict dietary control. Tryngolza is expected to be available in the U.S. before the end of 2024 and can be self-administered monthly via an auto-injector. This development not only signifies a major advancement for FCS patients but also highlights Ionis Pharmaceuticals’ growth as a biotechnology company.