FDA grants orphan drug designation to oral Huntington’s disease therapy

The FDA has granted orphan drug designation to an investigational oral therapeutic for the treatment of Huntington’s disease, according to the manufacturer.

Sage Therapeutics said in a press release that multiple clinical studies analyzing SAGE-718 for the treatment of cognitive disorders associated with NMDA receptor dysfunction are ongoing, including two placebo-controlled phase 2 studies and a phase 3 open-label safety study, as well as phase 2 placebo-controlled studies in mild cognitive impairment associated with Parkinson’s disease and MCI and mild dementia due to Alzheimer’s disease.

SAGE-718 previously received fast track designation from the FDA to treat Huntington’s disease and received orphan drug designation for Huntington’s disease from the European Medicines Agency.

“There are currently no approved treatments to address cognitive impairment for people with [Huntington’s disease] and a growing sense of urgency among researchers and people living with HD to address cognitive impairment early so that patients can maintain independence longer,” Laura Gault, MD, PhD, chief medical officer at Sage Therapeutics, said in the release.”