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Alixorexton Granted Breakthrough Therapy Designation by U.S. FDA for the Treatment of Narcolepsy Type 1

Alkermes plc (ALKS) | 2026-01-06

By Fiona Wilson

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Alkermes plc announced that the U.S. FDA granted Breakthrough Therapy designation to alixorexton for narcolepsy type 1 treatment, highlighting its potential as a novel treatment option.

Alixorexton, an oral selective orexin 2 receptor agonist, delivered promising results in phase 1 and phase 2 clinical trials, demonstrating clinically meaningful improvements in wakefulness for patients with narcolepsy type 1.

Following the successful phase 2 studies, Alkermes is preparing to advance alixorexton into phase 3 development to further evaluate its efficacy and safety profile.

Breakthrough Therapy Designation

The FDA's grant of Breakthrough Therapy status to alixorexton recognizes its potential as a significant improvement over existing narcolepsy type 1 treatments, expediting its regulatory review process.

Positive Clinical Data

Alixorexton's phase 2 trials displayed meaningful enhancements in wakefulness for narcolepsy type 1 patients, indicating a strong clinical benefit and safety profile for the drug.

Phase 3 Development

Alkermes is gearing up for phase 3 development of alixorexton to conduct further assessment of its efficacy and safety, a crucial step towards potential commercialization.

  • The Breakthrough Therapy designation for alixorexton by the FDA not only accelerates the regulatory pathway of the drug but also signifies its positioning as a promising and much-needed therapeutic option for narcolepsy type 1 patients.
  • The positive outcomes from the phase 2 trials reinforce the effectiveness and safety of alixorexton, paving the way for expanded clinical trials and eventual market entry as a breakthrough narcolepsy treatment.

Alkermes, with the FDA's Breakthrough Therapy designation for alixorexton, is strategically positioned to push the drug towards addressing the unmet medical needs in narcolepsy type 1 treatment, marking an important milestone in the company's pursuit of innovative healthcare solutions.