Alkermes has received orphan drug designations (ODD) from both the US Food and Drug Administration (FDA) and the European Commission (EC) for alixorexton, its investigational oral orexin 2 receptor (OX2R) agonist to treat idiopathic hypersomnia (IH) and narcolepsy.
The FDA granted the status to alixorexton for idiopathic hypersomnia while the EC approved the designation for narcolepsy.
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In the US, the ODD offers incentives such as tax credits for clinical testing, exemption from certain FDA application fees, and seven years of marketing exclusivity if approved.
In the EU, benefits include protocol support, reduced regulatory fees, and up to ten years of exclusivity upon approval.
Previously, the FDA also granted breakthrough therapy designation to alixorexton for narcolepsy type 1 (NT1).
The therapy is under evaluation in Phase III Brilliance Studies for NT1 and NT2 in adults, as well as in the Phase II Vibrance-3 study for idiopathic hypersomnia.
Narcolepsy, a rare and chronic neurological disorder, disrupts the regulation of the sleep-wake cycle.
Its primary symptom is excessive daytime sleepiness, and it is categorised by type based on the presence of loss of orexin neurons and cataplexy.
IH is another rare sleep disorder marked by pronounced daytime sleepiness despite adequate nighttime sleep. Symptoms include sleep inertia, fatigue, and cognitive issues.
Alixorexton (previously ALKS 2680) targets the orexin system, considered central to the regulation of wakefulness, for potential benefit in hypersomnolence disorders regardless of orexin deficiency.
Alkermes chief medical officer Craig Hopkinson said: “These orphan drug designations represent important milestones for the alixorexton programme and underscore its potential, if approved, to advance care for the narcolepsy and idiopathic hypersomnia patient communities.
“Alixorexton’s Phase II clinical trial results in NT1 and NT2 underscore its potential to become a differentiated treatment option. We look forward to continuing our momentum in the alixorexton development programme as we enrol the Phase III Brilliance Studies and work to complete the Vibrance-3 Phase II study in IH this year.”
