Alkermes (NASDAQ:ALKS) announced on January 6, 2026, that the U.S. FDA has granted Breakthrough Therapy Designation to alixorexton (formerly ALKS 2680) for the treatment of narcolepsy type 1 (NT1).
This designation is based on compelling data from the Phase 2 Vibrance-1 study, which showed that the oral orexin 2 receptor (OX2R) agonist significantly improves wakefulness and stabilizes the sleep-wake cycle in patients with severe symptoms.
The Breakthrough Therapy status is intended to fast-track the development of drugs that show a substantial improvement over existing treatments for serious conditions.
For NT1 patients—who suffer from a lack of natural orexin-producing neurons—alixorexton acts as a "replacement therapy" by directly stimulating the OX2R.
This mechanism addresses the underlying biological cause of the disorder, rather than just masking symptoms like traditional stimulants.
In the Vibrance-1 trial (n=92), alixorexton demonstrated statistically significant and dose-dependent improvements in mean sleep latency.
At baseline, patients fell asleep in roughly 3 minutes during the Maintenance of Wakefulness Test (MWT); after treatment, they achieved "normative wakefulness" (≥20 minutes), with the 8 mg dose group reaching an average of 28 minutes.
Beyond wakefulness, the drug also showed robust improvements in patient-reported outcomes for fatigue and cognitive function, with a generally well-tolerated safety profile