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# Rare Pediatric Disease Status for Gaboxadol
- URL: https://www.fdaweb.com/rare-pediatric-disease-status-for-gaboxadol/
- Published: 2020-06-19T12:00:00.000Z
- Updated: 2026-09-14T16:18:51.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5147054

FDA has granted Ovid Therapeutics a rare pediatric disease designation to OV101 (gaboxadol) for treating Angelman syndrome. The company says that OV101 is believed to be the only delta-selective GABA receptor agonist in development and is currently being evaluated in the Phase 3 NEPTUNE trial, with topline results expected in the fourth quarter. The agency previously granted orphan drug and fast track designations for OV101 for the same indication.

Angelman syndrome is a rare genetic condition that is characterized by a variety of signs and symptoms, such as delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety, Ovid says. The most common cause of Angelman syndrome is a nonfunctioning gene that codes for ubiquitin protein ligase E3A, which plays a critical role in nerve cell communication, it says. Individuals with Angelman syndrome typically have normal lifespans but are unable to live independently.