European Medicines Agency (EMA) has granted Orphan Drug Designation to biopharmaceutical company Sage Therapeutics’ SAGE-718 for the treatment of Huntington’s disease (HD).
SAGE-718 is a first-in-class NMDA receptor positive allosteric modulator (PAM) as well as lead neuropsychiatric drug candidate.
It is in development as a potential oral therapy for cognitive disorders related to NMDA receptor dysfunction, potentially including HD, Parkinson’s disease (PD) and Alzheimer’s disease (AD).
Several clinical studies are ongoing with SAGE-718 across many disease areas.
These include two placebo-controlled Phase 2 trials and a Phase 3 open-label safety study in the lead indication of HD-related cognitive impairment, as well as additionally Phase 2 placebo-controlled studies in mild cognitive impairment (MCI) related to PD and MCI and mild dementia due to AD).
Sage Therapeutics chief medical officer Laura Gault said: “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.
“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.”