FDA names SAGE-718 orphan drug for Huntington’s disease

The therapy was granted fast-track status in 2021

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SAGE-718, Sage Therapeutics’ experimental oral therapy for Huntington’s disease.

The FDA gives this designation to therapies that have the potential to treat rare diseases, which are defined as conditions that affect fewer than 200,000 people in the U.S. The designation is designed to incentivize developing treatments where there might not be a strong economic incentive to make an investment.

Therapies named orphan drugs are guaranteed no competition from generics or other formulations for seven years in U.S. markets, if they’re approved.

The designation “provides continued momentum in our efforts to help patients and their families impacted by this aspect of HD [Huntington’s disease],” Laura Gault, MD, PhD, Sage’s chief medical officer, said in a company press release.

SAGE-718 was granted FDA’s fast-track designation for treating Huntington’s in 2021. This is designed to speed the development and review of potentially important new treatments. The therapy was designated an orphan drug by the European Medicines Agency for the same indication earlier this year.

“Cognitive impairment is one of the most underrecognized aspects of HD,” Gault said. “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.”

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What is SAGE-718?

SAGE-718 is an orally available molecule that’s intended to improve cognitive function by modulating the activity of N-methyl-D-aspartate (NMDA), a brain protein involved in learning and memory. Its impaired activity is thought to contribute to nerve cell dysfunction in Huntington’s and other neurological disorders.

The safety and effects on cognitive function of SAGE-718, given once a day, are being assessed against a placebo in people with Huntington’s and cognitive impairments in two Phase 2 clinical trials — DIMENSION (NCT05107128) and SURVEYOR (NCT05358821).

Conducted at sites in the U.S., U.K., Canada, and Australia, DIMENSION involves people with genetically confirmed Huntington’s who haven’t developed symptoms or are at the early stages of the disease. SURVEYOR includes early patients and healthy people in the U.S. Both studies may still be recruiting up to a total of 268 participants, ages 25-65.

An ongoing Phase 3 study called PURVIEW (NCT05655520) is testing SAGE-718’s long-term safety and tolerability in Huntington’s patients who complete either of the Phase 2 studies, as well as other patients. It’s taking place in the U.S. and Canada, and recruitment is likely ongoing.

Sage is also running studies to test SAGE-718 as a treatment for Parkinson’s disease and Alzheimer’s disease.