CAMBRIDGE, Mass.– Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) 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 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. For more information about orphan designation, please visit the FDA website at www.fda.gov.
SAGE-718 previously received Fast Track Designation from the FDA for HD, and orphan drug designation for HD by the European Medicines Agency.
About SAGE-718
SAGE-718, a first-in-class investigational NMDA receptor positive allosteric modulator (PAM), is in development as a potential oral therapy for cognitive disorders associated with NMDA receptor dysfunction, including HD, PD and AD. Sage is advancing a clinical program for SAGE-718 with multiple ongoing placebo-controlled Phase 2 studies across multiple disease areas, including its potential lead indication, cognitive impairment associated with HD, as well as cognitive impairment due to AD and PD. The Company is also conducting a Phase 3 open-label safety study in HD cognitive impairment.
About Huntington’s disease and cognition
HD is a rare, inherited neurodegenerative disease that progresses over time and affects up to an estimated 40,000 adults in the U.S. each year. Cognitive impairment can severely affect people with HD. There are currently no treatment options available to improve the cognitive effects of the disease.
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.
Contact
Matthew Henson
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