Pharma Focus America

Sage Therapeutics Secures Orphan Drug Designation from FDA for SAGE-718 in the Treatment of Huntington's Disease

Thursday, October 19, 2023

Sage Therapeutics, Inc. (Nasdaq: SAGE) is a biopharmaceutical company that is committed to improving brain health. They have recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to their drug, SAGE-718, for the treatment of Huntington’s disease (HD). SAGE-718 is currently in development as a potential oral therapy for cognitive disorders associated with dysfunction in the NMDA receptor. Multiple clinical studies are currently underway for SAGE-718, including two placebo-controlled Phase 2 trials, as well as a Phase 3 open-label safety study for HD-related cognitive impairment. In addition, there are Phase 2 placebo-controlled studies being conducted for mild cognitive impairment (MCI) associated with Parkinson's disease (PD) and MCI and mild dementia due to Alzheimer's disease (AD).

Sage Therapeutics, emphasized the devastating impact of Huntington’s disease, particularly on cognitive function, which is often overlooked. She noted the absence of approved treatments to address cognitive impairment in HD patients and the urgent need to tackle this aspect early to help patients maintain their independence. The FDA's Orphan Drug Designation is a significant step forward in their mission to assist HD patients and their families.

Orphan drug designation is granted by the FDA's Office of Orphan Products Development to incentivize the development of safe and effective therapies for rare diseases. It offers benefits such as grant funding for clinical trials, tax advantages, FDA user-fee benefits, and potential market exclusivity in the United States for seven years following FDA approval. However, it does not alter the standard regulatory requirements for drug approval. For more information on orphan designation, you can visit the FDA website.

SAGE-718 had previously received Fast Track Designation from the FDA for HD and orphan drug designation for HD from the European Medicines Agency.

SAGE-718 is a first-in-class investigational NMDA receptor positive allosteric modulator (PAM) under development as a potential oral therapy for cognitive disorders linked to NMDA receptor dysfunction in conditions like HD, PD, and AD. Sage Therapeutics is running multiple placebo-controlled Phase 2 studies, focusing on cognitive impairment associated with HD, AD, and PD. They are also conducting a Phase 3 open-label safety study for HD-related cognitive impairment.

Huntington's disease is a rare, hereditary neurodegenerative condition that affects approximately 40,000 adults in the U.S. each year. Cognitive impairment is a significant issue in HD, and currently, there are no approved treatments to address these cognitive effects.



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