Sage Therapeutics, Inc. announced the European Medicines Agency (EMA) granted Orphan Drug Designation to SAGE-718 for the treatment of Huntington’s disease (HD). SAGE-718 is in development as a potential oral therapy for cognitive disorders associated with NMDA receptor dysfunction. 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 lead indication of HD-related cognitive impairment, and additionally 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).
“Huntington’s disease is a serious, debilitating condition that interferes with daily functioning in the prime years of life and is associated with significant morbidity and early mortality, and yet there are currently no approved therapies for the treatment of HD-related cognitive impairment,” said Laura Gault, M.D., Ph.D., Chief Medical Officer, Sage Therapeutics. “With this exciting development of Orphan Drug Designation from the EMA, we have further momentum in our efforts to address this unmet need. Our goal with SAGE-718 is to provide rapid, meaningful, and sustained improvement in cognitive functioning early in the disease so that patients can maintain independence longer.”
Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases that meet certain specified criteria. Potential benefits for Sage as a result of this designation include Protocol Assistance or scientific advice specifically designed for orphan medicines, European centralized authorization procedure, if development is successful and a marketing authorization application is filed, and if approved, ten years of market exclusivity and protection from similar medicines with the same indication being approved.