Alkermes Secures FDA Breakthrough Therapy Status for Alixorexton in Narcolepsy Type 1
Alkermes plc has announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to alixorexton for the treatment of narcolepsy type 1 (NT1).
The designation is based on phase 1 and phase 2 clinical data, including positive outcomes from Vibrance-1, a phase 2 study of 92 patients with NT1.
Alixorexton is an investigational, oral, selective orexin 2 receptor (OX2R) agonist under development for NT1, narcolepsy type 2 (NT2) and idiopathic hypersomnia.
The FDA’s Breakthrough Therapy designation is intended to accelerate the development and review of medicines for serious conditions that may offer significant improvement over existing therapies.
In Vibrance-1, alixorexton achieved the primary endpoint across all tested doses, showing statistically significant, dose-dependent improvements in wakefulness on the Maintenance of Wakefulness Test (MWT) compared with placebo. The treatment was generally well tolerated.
Alkermes plans to begin a global phase 3 development programme for alixorexton in NT1 in the first quarter of 2026.
If approved, the medicine has the potential to offer a new standard of care for patients with narcolepsy type 1 by targeting the orexin pathway and addressing the underlying causes of excessive daytime sleepiness.
