Ionis Rare Disease Drug Is Headed to FDA, But Forthcoming Data Will Decide Competitive Profile

MedCity News January 22, 2024
Frank Vinluan

In its Phase 3 test, Ionis Pharmaceuticals drug donidalorsen reduced the frequency of swelling attacks caused by the rare disease hereditary angioedema. If Ionis can commercialize this drug, competition would include products from Takeda Pharmaceutical and BioCryst Pharmaceuticals.

An Ionis Pharmaceuticals drug has preliminary data showing it met the main goal of its pivotal test in a rare disease with only a handful of approved treatments. With these results in hand, Ionis is now preparing to submit its drug candidate for FDA review. However, the key data that will determine how competitive the drug could be won’t become available until later this year.

The Ionis drug, donidalorsen, is in development for hereditary angioedema (HAE), an inherited disease that leads to...

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