September 9, 2025
Ionis Pharmaceuticals, Inc. today announced that it has received U.S. FDA Breakthrough Therapy Designation for ION582, as a treatment for Angelman syndrome.
According to the US FDA, a “Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).”
Read the press release.
Learn more about FDA Breakthrough Therapy designation.
