Ultragenyx Pharmaceutical Receives Breakthrough Therapy Designation from FDA for GTX-102

Ultragenyx Pharmaceutical Inc. 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).”

The release also revealed the official name of GTX-102 – apazunersen.

Read the press release. 

Learn more about the Breakthrough Therapy designation.