Ultragenyx Pharmaceutical Inc. today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GTX-102 (apazunersen) 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 the Breakthrough Therapy designation