Dublin — Alkermes plc said alixorexton, its investigational oral drug being developed for narcolepsy and idiopathic hypersomnia, has received orphan drug designations from regulators in the United States and Europe.
The U.S. Food and Drug Administration granted orphan drug designation to alixorexton for the treatment of idiopathic hypersomnia, while the European Commission granted orphan designation for the treatment of narcolepsy.
Alixorexton is a novel, selective orexin 2 receptor agonist being studied for the treatment of narcolepsy type 1, narcolepsy type 2 and idiopathic hypersomnia.
“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,” said Craig Hopkinson, M.D., (MBChB), Chief Medical Officer and Executive Vice President, Research & Development at Alkermes. “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.”
Orphan drug designation is intended to support the development of medicines for rare diseases. In the United States, the designation can provide incentives such as tax credits for qualified clinical testing, exemptions from certain FDA application fees and seven years of market exclusivity if the drug is approved. In the European Union, orphan designation may provide incentives including protocol assistance, reduced regulatory fees and up to 10 years of market exclusivity if approved.
Alixorexton previously received FDA Breakthrough Therapy designation for the treatment of narcolepsy type 1. The drug is currently being evaluated in the phase 3 Brilliance Studies in adults with narcolepsy type 1 and type 2, as well as in the phase 2 Vibrance-3 study in adults with idiopathic hypersomnia.
Narcolepsy is a rare, chronic neurological disorder that affects the brain’s ability to regulate the sleep-wake cycle. Excessive daytime sleepiness is the hallmark symptom, while other symptoms can include sleep paralysis, sleep-related hallucinations and disturbed nighttime sleep.
Narcolepsy type 1 is characterized by the loss of orexin-producing neurons and is associated with cataplexy, a sudden loss of muscle control while a person is awake, often triggered by strong emotions. The underlying cause of narcolepsy type 2 has not been fully established, though the orexin pathway may play an important role.
