Sage Therapeutics Granted Orphan Drug Designation for Huntington’s Disease Treatment
Biopharmaceutical company, Sage Therapeutics (Nasdaq: SAGE) has been granted Orphan Drug Designation for SAGE-718 for the treatment of Huntington’s disease (HD) by the FDA. SAGE-718 previously received Fast Track Designation from the FDA for HD, and orphan drug designation for HD by the European Medicines Agency.
Laura Gault, M.D., Ph.D., Chief Medical Officer, Sage Therapeutics, said, “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. 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.”
SAGE-718 is a first-in-class investigational NMDA receptor positive allosteric modulator (PAM) in development as a potential oral therapy for cognitive disorders associated with NMDA receptor dysfunction, including HD, PD and AD.
Sage is managing multiple ongoing clinical studies 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).
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.
About Sage Therapeutics
Sage Therapeutics is a biopharmaceutical company fearlessly leading the way to create a world with better brain health. Our mission is to pioneer solutions to deliver life-changing brain health medicines, so every person can thrive. For more information, please visit http://www.sagerx.com