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# Sage Huntington’s Drug Gets Orphan Status
- URL: https://www.fdaweb.com/sage-huntingtons-drug-gets-orphan-status/
- Published: 2023-10-31T12:00:00.000Z
- Updated: 2026-09-14T14:14:51.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5155685

FDA has granted Sage Therapeutics an orphan drug designation for SAGE-718 and its use in treating Huntington’s disease (HD). SAGE-718 is described as a first-in-class investigational NMDA receptor-positive allosteric modulator (PAM) being developed as a potential oral therapy for cognitive disorders associated with NMDA receptor dysfunction, including HD, Parkinson’s disease and Alzheimer’s disease.

“Multiple clinical studies are ongoing with SAGE-718 across several disease areas, including two placebo-controlled Phase 2 studies and a Phase 3 open-label safety study in the potential lead indication of HD-related cognitive impairment, as well as Phase 2 placebo-controlled studies in mild cognitive impairment (MCI) associated with Parkinson’s disease (PD) and MCI and mild dementia due to Alzheimer’s disease (AD),” the company says.

HD is described as a rare, inherited neurodegenerative disease that progresses over time and affects about 40,000 adults in the U.S. each year. Cognitive impairment can severely affect HD patients, Sage says, adding that there are currently no treatment options available to improve the cognitive effects of the disease.