Ionis Pharma Advances Olezarsen Towards Launch for Rare Disease Treatment Following Promising Trial Results

Ionis Pharma announced its progress with olezarsen, an investigational compound for treating familial chylomicronemia syndrome (FCS), a rare disease causing dangerously high triglyceride levels and potentially fatal pancreatitis. Following positive Phase 3 results, Ionis is preparing for a U.S. launch by year-end, pending regulatory approval. Olezarsen, which has received breakthrough and orphan drug designations, demonstrated significant reductions in triglycerides and a protein regulating them, alongside a meaningful decrease in acute pancreatitis events during the trial. This study is the first to show that lowering triglyceride levels can significantly reduce acute pancreatitis occurrences, positioning olezarsen as a potential standard care for FCS. Furthermore, preliminary results from a Phase 2b trial suggest olezarsen could also benefit patients with severe hypertriglyceridemia. Ionis is re-hiring key personnel and building support teams to aid patients throughout their treatment, indicating robust commercialization and patient care strategies in anticipation of the drug’s launch.

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[Source: MM+M, April 9th, 2024]