New Drug for Narcolepsy: Why Patients in Germany Must Continue to Wait

Falling asleep in the middle of a conversation or suddenly losing control of one’s muscles whilst laughing: for people with narcolepsy, this can be part of everyday life. Existing medicines merely alleviate the symptoms of this neurological condition. However, a new active ingredient could revolutionise treatment. Oveporexton has already been approved in the US, China and Japan. In Europe, however, those affected must continue to wait. Prof. Dr Ulf Kallweit, holder of the endowed professorship for Narcolepsy and Hypersomnolence Research at Witten/Herdecke University (UW/H), believes it may become available in Germany by 2027.

Targeting the cause for the first time

People with type 1 narcolepsy lack the neurotransmitter orexin, which stabilises wakefulness in the brain. Whilst existing medicines primarily tackle individual symptoms, Oveporexton mimics the effect of the missing neurotransmitter and activates the orexin system. “Orexin acts like a conductor of the nervous systems that promote wakefulness,” explains Kallweit.

The results of clinical trials are very promising: those affected remained awake for significantly longer and experienced sudden muscle weakness (‘cataplexy’) less frequently. “In the trials, people describe feeling normally awake again. The symptoms sometimes disappear completely or are greatly reduced whilst the drug is active,” says Kallweit. For sufferers, this could mean a daily life with significantly fewer limitations.

Can those affected access the medicine sooner?

Kallweit attributes the fact that oveporexton is not yet authorised in Europe, amongst other things, to additional requirements from the European Medicines Agency (EMA). The EMA has requested further data and an additional trial. Nevertheless, the researcher is confident: “The evidence is excellent.”

He also sees a possibility for severely affected people to receive the medicine earlier. So-called ‘hardship case’ programmes in Germany allow the use of medicines that have not yet been authorised, subject to certain conditions. 

“Particularly in the case of therapies that offer a significant improvement over existing treatment options, rapid access should be made possible,” emphasises Kallweit.

Hope for other conditions too

Oveporexton could be just the beginning. As orexin also influences attention, drive and metabolism, researchers are investigating possible applications for ADHD and severe daytime sleepiness, for example in connection with Parkinson’s disease. Its use for depression is also under discussion.

However, long-term data on efficacy and safety are still lacking. It remains to be seen how oveporexton will perform in everyday clinical practice. For Kallweit, the results so far nevertheless represent a significant step forward: for the first time, a treatment could enable people with type 1 narcolepsy to maintain near-normal alertness for much of the day.