Ionis plans NDA filing for first independent drug after positive data readout in rare genetic disorder
Ionis is another step closer to the launch of its first independent drug, touting positive Phase III data for olezarsen in patients with a rare genetic disorder called familial chylomicronemia syndrome (FCS).
Ionis said Tuesday it has plans to file for an NDA in early 2024 and for approvals in the EU after olezarsen hit its primary endpoint in the BALANCE study: a statistically significant reduction in triglyceride levels after six months on 80 mg of the drug compared to placebo, earning a p-value of 0.0009. A key secondary endpoint also showed promise, with olezarsen reducing acute pancreatitis events by 100%. In the placebo arm, there were 11 instances of pancreatitis.
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