RARE Daily

FDA Grants Fast Track Designation to Sage for Huntington’s Disease Therapy

September 15, 2021

The U.S. Food and Drug Administration granted Fast Track designation to Sage Therapeutics’ SAGE-718 for development as a potential treatment for Huntington’s disease.

Photo: Jim Doherty, chief research officer at Sage Therapeutics

Huntington’s disease (HD) is a rare, inherited neurodegenerative disease that progresses over time. Up to 30,000 adults are diagnosed with HD in the United States each year. Symptoms usually appear between ages 30–45, worsen over the following 15–20 years, and ultimately lead to death. Psychiatric and cognitive symptoms can severely affect people with HD.

“We believe that improving cognitive function is one of the core paths to maintaining quality of life in HD and remains an area of significant unmet medical need. In studies to date, treatment with SAGE-718 has been associated with improved cognitive performance, particularly in the domain of executive functioning,” said Jim Doherty, chief research officer at Sage Therapeutics. “The FDA Fast Track Designation is an important milestone in the development of SAGE-718, as it provides opportunities to engage collaboratively with the FDA to further clinical development and future regulatory review of SAGE-718 for the treatment of HD.”

Sage’s lead neuropsychiatric candidate, SAGE-718, is a first-in-class NMDA receptor PAM in development as a potential oral therapy for cognitive disorders associated with NMDA receptor dysfunction, potentially including Huntington’s disease, Parkinson’s disease, and Alzheimer’s disease. Ongoing studies aim to evaluate whether SAGE-718 may have the potential to improve cognitive symptoms for these difficult-to-treat disorders. Sage expects to initiate a placebo-controlled phase 2 trial with SAGE-718 in early to moderate HD in late 2021.

Fast Track is a process designed to facilitate the development, and expedite the review, of drugs to treat serious conditions and fill an unmet medical need. Drugs that receive Fast Track designation may be eligible to be the subject of more frequent communications and meetings with FDA to review the drug’s development plan including the design of the proposed clinical trials, use of biomarkers and the extent of data needed for approval. Drugs with Fast Track Designation may also qualify for priority review to expedite the FDA review process if relevant criteria are met.

Author: Rare Daily Staff

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