The designation for the investigational oral orexin 2 receptor agonist aims to expedite development based on positive phase 2 clinical trial data.
Key takeaways:
- The FDA granted Breakthrough Therapy designation to alixorexton for the treatment of narcolepsy type 1.
- The designation is based on phase 1 and 2 clinical data, including the Vibrance-1 study, which showed dose-dependent improvements in wakefulness.
- Alixorexton is an investigational, oral, selective orexin 2 receptor agonist also in development for narcolepsy type 2 and idiopathic hypersomnia.
- Alkermes, the drug’s developer, plans to initiate a global phase 3 program in the first quarter of 2026.
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to alixorexton for the treatment of narcolepsy type 1 (NT1). The designation was granted to Alkermes plc for its novel, investigational, oral, selective orexin 2 receptor (OX2R) agonist based on data from phase 1 and phase 2 studies.
The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious conditions where preliminary clinical evidence suggests the drug may demonstrate substantial improvement over available therapy.
“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,” says Craig Hopkinson, MD, chief medical officer and executive vice president of research & development at Alkermes, in a release. “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.”
The designation was supported by positive results from the Vibrance-1 phase 2 study. In the study, alixorexton met its primary endpoint across all doses, showing statistically significant and clinically meaningful improvements from baseline in wakefulness on the Maintenance of Wakefulness Test (MWT) compared to placebo. The drug was generally well-tolerated at all doses tested.
Alixorexton targets the orexin system, which is considered a primary regulator of wakefulness. The drug is also in development for the treatment of narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH).
Alkermes plans to initiate the alixorexton global phase 3 narcolepsy program in the first quarter of 2026.
