DUBLIN — Alkermes plc said the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to alixorexton for the treatment of narcolepsy type 1, based on phase 1 and phase 2 clinical data, including positive results from the Vibrance-1 phase 2 study.
Alixorexton is a novel, investigational, oral, selective orexin 2 receptor agonist being developed for narcolepsy type 1, narcolepsy type 2, and idiopathic hypersomnia. The Vibrance-1 trial was a phase 2 study involving 92 patients with narcolepsy type 1.
The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of drugs that treat serious conditions and show preliminary clinical evidence of substantial improvement over existing therapies on clinically significant endpoints.
“Alixorexton may offer substantial improvements over available therapy for people living with narcolepsy type 1, a community that has continued to face profound unmet medical needs despite available treatments. This Breakthrough Therapy designation underscores the strength of alixorexton’s initial clinical data and supports our conviction that targeting the orexin pathway has the potential to fundamentally shift treatment expectations for central disorders of hypersomnolence. If approved, alixorexton’s differentiated profile and compelling efficacy may represent a new standard of care in narcolepsy type 1. We look forward to working closely with the FDA as we plan to advance alixorexton into phase 3 development later this quarter,” said Craig Hopkinson, M.D., Chief Medical Officer and Executive Vice President of Research & Development at Alkermes.
In the Vibrance-1 study, alixorexton met the primary endpoint across all doses tested, demonstrating statistically significant, clinically meaningful, and dose-dependent improvements from baseline compared with placebo in wakefulness as measured by the Maintenance of Wakefulness Test in patients with narcolepsy type 1. The drug was generally well tolerated at all doses evaluated.
Alkermes said it plans to initiate a global phase 3 clinical program for alixorexton in narcolepsy in the first quarter of 2026. (Source: IANS)
