SAGE-718 for Huntington’s Disease Earns Orphan Drug Designation


Currently, there are no FDA-approved treatments designed for cognitive impairment related to Huntington’s disease. However, to improve the lives of those affected, additional research and therapeutic development are urgently needed.

According to an article in Healio, Sage Therapeutics aims to address this unmet need through the development of SAGE-718. The ALZForum describes SAGE-718 as:

“a derivative of the endogenous steroid 24(S)-hydroxycholesterol. It is a positive allosteric modulator of the NMDA receptor, whose activity induces long-term potentiation of synapses and is thus essential for learning and memory.”

Recently, the FDA granted Orphan Drug designation to this investigational treatment. This status is granted to drugs or biologics intended to treat, diagnose, or prevent rare conditions. In the United States, conditions are considered rare if they affect fewer than 200,000 people. Orphan Drug status comes with incentives such as fee waivers, tax credits, and seven years of market exclusivity if the drug is approved.

Research into SAGE-718 is still ongoing. It is currently being evaluated in multiple clinical studies for conditions such as mild cognitive impairment, Parkinson’s disease, and Alzheimer’s disease.

About Huntington’s Disease (HD)

Huntington’s disease is a rare, progressive neurodegenerative disorder. Progressive means that it worsens over time. This disease is caused by HTT mutations, which lead to abnormal and toxic buildups of huntingtin protein, resulting in neuronal death. Huntington’s disease is extremely rare in children but is more severe in this group. More often, Huntington’s disease manifests in one’s 30s and 40s, with an expected lifespan of 15-20 years following diagnosis.

Currently, there is no cure, and no way to reverse symptoms. However, treatments can help reduce involuntary movements called chorea. Other symptoms include insomnia, lethargy, seizures, chest infections, dystonia, tremors, cognitive decline, slurred speech, difficulty walking, problems with balance and coordination, unexplained weight loss, dysphagia (difficulty swallowing), and changes in mood, behavior, and personality.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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