A treatment in sight for narcolepsy

Wake-up call

Narcolepsy is a chronic brain disorder that affects around 3 million people worldwide. It disrupts the brain’s ability to control sleep and wakefulness, leading to fragmented nighttime slumber and intense daytime fatigue.

People with narcolepsy type 1 also experience cataplexy, a sudden loss of muscle strength triggered by strong emotions such as anger or excitement. In severe cases, this can cause people to collapse.

There is no cure for narcolepsy. Current treatments focus on managing symptoms, often with a combination of medications. Stimulants are used to help patients stay awake during the day, while nighttime drugs known as oxybates, such as Jazz Pharmaceuticals’ Xywav, are prescribed to improve sleep. Antidepressants are also used to reduce episodes of sudden muscle weakness.

These treatments can help, but they come with trade-offs. Side effects can include heart risks, weight changes, insomnia and mental health issues, says Aaron Schokman, a sleep researcher at the University of Sydney who also lives with the condition.

Better approaches are on the horizon.

Takeda Pharmaceutical is preparing to seek global regulatory approval by March for a new drug that targets the root cause of narcolepsy rather than just its symptoms. The drug, oveporexton, is taken twice daily and belongs to a new class of medicines designed to replace orexin, a brain chemical that helps regulate wakefulness and is deficient in people with narcolepsy type 1.

“This new class of treatments is a game changer,” Schokman says. “There’s a lot of promise in it.”

Unlike current therapies, orexin-based drugs are aimed at restoring the brain’s natural wake-up signal. Schokman says longer-term data will be needed to determine whether a single treatment can replace today’s multi-drug approach. Still, he says, for many patients, the prospect of fewer medications and fewer side effects offers real hope.

Takeda is also developing another candidate, TAK-360, for idiopathic hypersomnia, or excessive daytime sleepiness.

The potential impact of these medications could be large. As many as 20% of people worldwide suffer from excessive daytime sleepiness. — Kanoko Matsuyama