Ionis Pharmaceuticals plans to file for NDA for its first independent drug after positive Phase III data in a rare genetic disorder
Ionis Pharmaceuticals, a leading biopharmaceutical company, is one step closer to launching its first independent drug. The company has announced positive Phase III data for its drug olezarsen in patients with a rare genetic disorder known as familial chylomicronemia syndrome (FCS). Ionis plans to file for a New Drug Application (NDA) in early 2024 and seek approvals in the European Union (EU).
Promising results from the BALANCE study
Ionis has reported that olezarsen met its primary endpoint in the BALANCE study, showing a statistically significant reduction in triglyceride levels after six months of treatment with 80 mg of the drug compared to placebo. The study demonstrated a p-value of 0.0009, indicating strong efficacy of the drug. Additionally, a key secondary endpoint showed promise, as olezarsen reduced the occurrence of acute pancreatitis events by 100%. In the placebo arm of the study, 11 instances of pancreatitis were observed.
Next steps for Ionis Pharmaceuticals
With the positive Phase III data in hand, Ionis plans to pursue regulatory approval for olezarsen. The company intends to file for an NDA in early2024, seeking market authorization to make the drug available to patients with FCS. Ionis also aims to obtain approvals in the EU, expanding access to the drug to a broader patient population. This milestone represents a significant step forward for Ionis as it strives to bring its first wholly-owned drug to market.
Keywords: Ionis Pharmaceuticals, independent drug, Phase III data, genetic disorder, familial chylomicronemia syndrome, NDA, triglyceride levels, pancreatitis**