The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Sage Therapeutics’ 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 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).
“Huntington’s disease is a devastating condition that often affects patients in their prime years, and it can significantly impact a patient’s ability to live independently. Cognitive impairment is one of the most underrecognized aspects of this disease,” said Laura Gault, M.D., Ph.D., Chief Medical Officer, Sage Therapeutics.
“There are currently no approved treatments to address cognitive impairment for people with HD 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. The ODD designation from the FDA provides continued momentum in our efforts to help patients and their families impacted by this aspect of HD.”
Orphan drug designation is granted by the FDA Office of Orphan Products Development to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders.
Under the Orphan Drug Act, the FDA may provide grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and the potential for seven years of market exclusivity in the United States for the drug in the orphan indication following drug approval by the FDA.
The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval.
SAGE-718 previously received Fast Track Designation from the FDA for HD, and orphan drug designation for HD by the European Medicines Agency.