The investigational oral orexin 2 receptor agonist received designations from the FDA for idiopathic hypersomnia and the European Commission for narcolepsy.
Key takeaways:
- The US FDA granted orphan drug designation to alixorexton for the treatment of idiopathic hypersomnia, while the European Commission granted the designation for narcolepsy.
- Alixorexton is currently being evaluated in phase 3 clinical trials for narcolepsy type 1 and type 2, and a phase 2 study for idiopathic hypersomnia.
- Orphan drug designation provides development incentives, including potential market exclusivity of seven years in the US and up to 10 years in the European Union, if approved.
Regulatory bodies in the United States and Europe recently granted alixorexton, an investigational, oral, selective orexin 2 receptor (OX2R) agonist in development by Alkermes plc for the treatment of narcolepsy type 1 (NT1), narcolepsy type 2 (NT2), and idiopathic hypersomnia (IH), orphan drug designations from regulatory bodies in the US and Europe.
The US Food and Drug Administration (FDA) granted the designation to alixorexton for the treatment of IH, and the European Commission granted it for the treatment of narcolepsy. Alixorexton previously received breakthrough therapy designation for the treatment of NT1 from the FDA.
“Narcolepsy and idiopathic hypersomnia are rare, chronic neurological conditions for which significant unmet need remains. These orphan drug designations represent important milestones for the alixorexton program and underscore its potential, if approved, to advance care for the narcolepsy and idiopathic hypersomnia patient communities,” says Craig Hopkinson, MD, MBChB, chief medical officer and executive vice president, research & development at Alkermes, in a release. “Alixorexton’s phase 2 clinical trial results in narcolepsy type 1 and type 2 underscore its potential to become a differentiated treatment option. We look forward to continuing our momentum in the alixorexton development program as we enroll the phase 3 Brilliance Studies and work to complete the Vibrance-3 phase 2 study in IH this year.”
Alixorexton is currently being evaluated in the phase 3 Brilliance Studies in adults with NT1 and NT2, and in the phase 2 Vibrance-3 study in adults with IH.
Orphan drug designation supports the development of medicines intended to treat rare diseases. In the US, the designation qualifies the drug developer for development incentives, including tax credits for qualified clinical testing, exemptions from certain FDA application fees, and seven years of market exclusivity, if approved. In the European Union, it provides incentives that may include protocol assistance, reduced regulatory fees, and up to 10 years of market exclusivity, if approved.
