GoKawiilPhysicians test new narcolepsy treatments by checking whether the drugs help people to fall asleep and stay asleep throughout the night.Credit: Lucia Graphus/Alamy
Earlier this month, the US Food and Drug Administration approved the first drug designed to treat the root cause of narcolepsy, a condition that affects millions of people by causing extreme sleepiness during the day and wakefulness at night.
‘Timer’ in the brain tracks sleep — and predicts awakening
The success of the drug, called oveporexton and marketed as Orzeyful, is being celebrated by those who struggle with narcolepsy. “It’s the best thing that has ever happened to me,” says Tyler Chapman, who volunteered for one of the clinical trials that contributed to the drug’s approval. But it’s also being watched with anticipation by researchers, who see it as the first in a line of therapies for sleep disorders that are likely to be approved in the coming years.
These emerging treatments, known as orexin agonists, are exciting because they promise not just to alleviate symptoms of narcolepsy but also to target the disease’s biological cause — and might even have implications for other neuropsychiatric disorders. With this new strategy against narcolepsy, pharmaceutical companies have jumped at the chance to tap into a market that is projected to top US$6.4 billion annually by the early 2030s.
“This is a tremendously exciting time for patients and for clinicians and scientists who have been working towards this for many years,” says Barry Lubarsky, the vice-president of medical affairs at the pharmaceutical company Alkermes, which is based in Dublin, Ireland, and is developing its own narcolepsy treatment.
A chance to feel normal
For Chapman, a 19-year-old student at the University of Tennessee, Knoxville, the milestone is life changing. Chapman had long struggled with his condition, at times sleeping for 16 hours a day, struggling in school and relying heavily on coffee. When he laughed, it would sometimes trigger his body to collapse, even though he remained fully conscious — a symptom known as cataplexy. Instead of experiencing all that university life has to offer, Chapman says that he rarely went out.
That is, until he volunteered earlier this year for a clinical trial with Takeda Pharmaceuticals America in Cambridge, Massachusetts, to test a new class of narcolepsy drug. He had previously tried more than a dozen medications, including stimulants, but often experienced severe psychological side effects.
A new class of narcolepsy drug mimics the neuropeptide orexin. Shown here is one form of orexin that has 28 amino acids.Credit: Laguna Design/Science Photo Library
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