A new treatment for narcolepsy has been shown to significantly improve some of the symptoms that most affect people living with this disease. The results, published in The New England Journal of Medicine, show improved ability to stay awake during the day, a substantial reduction in excessive daytime sleepiness, and fewer episodes of cataplexy, one of the most characteristic manifestations of the disease.
The study involved the participation of Alex Iranzo, Head of the Sleep Unit at Clínic Barcelona, leader of the Clinical Neurophysiology research group at IDIBAPS, and professor at the University of Barcelona. The researchers analyzed the results of two international clinical trials involving 273 people with narcolepsy.
A disease that disrupts the control of sleep and wakefulness
Narcolepsy is a chronic incurable neurological disorder that affects the brain's ability to regulate sleep and wakefulness. It is a rare disease affecting between 20 and 60 people per 100,000 inhabitants and usually begins during adolescence or early adulthood.
People with narcolepsy experience intense daytime sleepiness that can interfere with everyday activities such as studying, working, or driving. Many also experience cataplexy, sudden episodes of muscle weakness typically triggered by emotions such as laughter or surprise.
In the form of the disease associated with cataplexy, known as type 1 narcolepsy, there is a loss of the neurons that produce orexin, a substance essential for maintaining wakefulness. "Although treatments are available to help control some symptoms, until now none had acted on this biological mechanism," notes Alex Iranzo.
The treatment improves daytime sleepiness and the ability to stay awake
Researchers evaluated 273 people with narcolepsy in two independent international studies. Participants received either the treatment or placebo for 12 weeks and were assessed using objective tests and clinical questionnaires.
At the end of the treatment period, patients showed a marked improvement in their ability to stay awake during the day. Between 48% and 69% of treated patients achieved values considered normal in wakefulness tests, whereas virtually no participants in the placebo group achieved these results.
Daytime sleepiness also decreased significantly. Scores on the Epworth Sleepiness Scale, a tool used to evaluate this symptom, decreased by 9.7 to 11.8 points with treatment, compared with reductions of only 1.5 to 1.7 points with placebo. As a result, up to 84% of treated patients achieved sleepiness levels considered normal.
Fewer episodes of cataplexy and better quality of life
The treatment also substantially reduced the frequency of cataplexy episodes. Treated participants experienced reductions of between 79% and 89% in the weekly number of attacks.
Researchers also observed improvements in overall disease severity and in several quality-of-life indicators related to physical and mental health. Most treated participants reported a clear improvement in their symptoms by the end of the study.
"Until now, we had treatments that helped control some symptoms of narcolepsy, but this therapeutic approach acts on the biological mechanism that causes the disease," explains Alex Iranzo. "The results show simultaneous improvements in different manifestations of the disease, such as daytime sleepiness and cataplexy, and a significant proportion of patients reached values close to normality in some of the scales used to assess treatment response."
The results were consistent across both clinical trials and confirm the benefits previously observed in phase 2 studies, reinforcing the potential of this therapeutic strategy to simultaneously address several of the main symptoms of narcolepsy.
FDA approves the first treatment targeting the cause of narcolepsy
Since August 2026, the U.S. Food and Drug Administration (FDA) has approved this treatment for people with type 1 narcolepsy. It is the first approved therapy that acts on the biological mechanism responsible for the disease, representing a significant advance in a disorder for which available treatments had previously focused mainly on symptom control. The treatment has also been approved in China and Japan.
Study reference:
Dauvilliers Y, Mignot E, Antczak J, Arnulf I, Barateau L, Buntinx E, Corser B, Iranzo A, Khatami R, Lammers GJ, Lederer K, Nilsen KB, Pizza F, Schmidt MH, Sivam S, Sum-Ping O, Taniguchi M, Zhan S, Abraham A, Cai A, Du Y, Etherton MR, Faessel H, Hsiao S, Kadali H, Koundourakis E, Naylor M, Neuwirth R, Olsson T, Rogers R, Sheikh S, Tanaka S, von Rosenstiel P, von Hehn C, Hang Y, Yao B, Del Rio Villegas R, Plazzi G. Oveporexton for Narcolepsy Type 1 - Results from Two Phase 3 Trials. N Engl J Med. 2026 Sep 9. doi: 10.1056/NEJMoa2601598
