FDA Acts to Support Development of Ionis Therapy ION582

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease status to ION582, Ionis Pharmaceutical’s investigational treatment for Angelman syndrome. The announcement of the new FDA designations — both aimed at encouraging the therapy’s development — follows the launch of the…

OV101 Gets FDA’s Rare Pediatric Disease Status for Treating Angelman

Ovid Therapeutics‘ OV101 (gaboxadol) has been granted a rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of Angelman syndrome. This designation is…

Potential Angelman’s Treatment, GTX-102, Named Orphan Drug and Rare Pediatric Disease Therapy by FDA

A potential treatment for Angelman syndrome, GTX-102, has been granted both orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA) to support and help speed its advancement. The companies co-developing GTX-102 plan to file an investigational new drug application with the FDA in early 2020,…