Posted by Michael Wonder on 04 Sep 2019
GeneTx and Ultragenyx announce orphan drug designation and rare paediatric disease designation for GTX-102
3 September 2019 - GeneTx Biotherapeutics and Ultragenyx today announced that the U.S. FDA has granted orphan drug designation and rare paediatric disease designation to GTX-102 for the treatment of Angelman syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide.
GTX-102 is currently in late preclinical development, and an investigational new drug application is expected to be filed with the FDA in the first half of 2020.
In August 2019, Ultragenyx and GeneTx announced a partnership to develop GTX-102 with Ultragenyx receiving an exclusive option to acquire GeneTx.
