Ultragenyx receives Breakthrough Therapy Designation for GTX-102

Ultragenyx (RARE) Pharmaceutical announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for GTX-102 as a treatment for Angelman syndrome. “FDA Breakthrough Therapy Designation underscores both the urgent need for an effective treatment for patients and families affected by Angelman syndrome and the clinically meaningful results demonstrated to date with GTX-102,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “Based on the strength of the Phase 2 data and with strong support and interest from the Angelman syndrome community, our Phase 3 Aspire study is rapidly enrolling across our global sites. We look forward to advancing GTX-102 through the development process as rapidly as possible to bring this potential treatment to patients.”