Ionis Pharma’s genetic disease drug succeeds in late-stage study, ET HealthWorld


New Delhi: Ionis Pharmaceuticals said on Monday its drug for the treatment of a rare genetic disease met the main goal of a late-stage trial.

The drug, donidalorsen, was more successful than placebo in reducing the rate of attacks in patients with hereditary angioedema. They were treated with an 80 milligram dose through subcutaneous injection every four weeks or every eight weeks.

Hereditary angioedema is a rare and life-threatening genetic disease that causes unpredictable and frequent severe swelling of the skin, gastrointestinal tract, upper respiratory system, face and throat.

Based on the results of the study, Ionis is preparing to submit a marketing application with the U.S. Food and Drug Administration, the drugmaker said.

  • Published On Jan 22, 2024 at 06:52 PM IST

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