Approval Granted for Narcolepsy Drug in China
In China, patients receiving existing drug treatments for narcolepsy are not satisfied with their current options. This dissatisfaction is due to the limited efficacy and side effects of the available treatments, many of which are "off-label" usage and scheduled drugs with strict prescription regulations.
However, there is hope on the horizon. The Chinese guidelines for diagnosis and treatment for narcolepsy (2022) have listed Pitolisant (Wakix) as a first-line treatment therapy. Pitolisant, a selective histamine 3 (H3) receptor antagonist/inverse agonist, is the first approved innovative drug for narcolepsy and the only non-scheduled drug with narcolepsy indication in China. The NMPA has approved Pitolisant for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy.
The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have granted orphan drug designation for the treatment of narcolepsy to Pitolisant (Wakix), and the Chinese guidelines have emphasized that it is the first recommendation with level A evidence for EDS, cataplexy, hypnagogic hallucinations, and sleep paralysis.
Shawn Xiang, CEO of RareStone Group, stated that they are pleased to bring a new therapeutic option for Chinese narcolepsy patients. RareStone Group is working with Bioprojet to bring this promising treatment drug for narcolepsy to patients in China.
Besides RareStone Group and Tris Pharma, Takeda and Centessa Pharmaceuticals are currently working on treatments for narcolepsy in China. Takeda is developing Oveporexton, an oral orexin-2 receptor (OX2R) agonist that has shown positive results in two pivotal Phase 3 trials for narcolepsy type 1. Takeda is preparing to file for FDA and global regulatory approvals in 2025, with potential sales projected to reach $1 billion by 2030.
Centessa Pharmaceuticals is advancing an orexin-2 receptor agonist named ORX750, which is in Phase 2a clinical trials for narcolepsy types 1 and 2, as well as idiopathic hypersomnia. Phase 1 data showed promising results in improving wakefulness and sleep latency metrics. Centessa plans to proceed rapidly toward registrational studies.
More than half of narcolepsy patients in China have experienced misdiagnosis, highlighting the urgent need for better and more accessible treatments. RareStone Group, Tris Pharma, Takeda, and Centessa Pharmaceuticals are working to address this need, aiming to bring more accessible and affordable treatment options to rare disease patients in China.
Narcolepsy is a rare, chronic, debilitating neurological disorder of sleep-wake state instability, primarily characterized by excessive daytime sleepiness (EDS), cataplexy, and nocturnal sleep disturbances. With these new treatment options on the horizon, patients in China may soon find relief from the symptoms of this challenging condition.
[1] Takeda Pharmaceutical Company Limited. (2022). Takeda's investigational treatment for narcolepsy type 1 demonstrates positive results in two pivotal Phase 3 trials. Retrieved from https://www.takeda.com/news/press-releases/2022/takedas-investigational-treatment-for-narcolepsy-type-1-demonstrates-positive-results-in-two-pivotal-phase-3-trials/
[2] Centessa Pharmaceuticals. (2022). Centessa Pharmaceuticals announces positive data from Phase 1 clinical trial of ORX750 in narcolepsy and idiopathic hypersomnia. Retrieved from https://www.centessapharma.com/news-and-media/press-releases/detail/151/centessa-pharmaceuticals-announces-positive-data-from-phase-1-clinical-trial-of-orx750-in-narcolepsy-and-idiopathic-hypersomnia
[3] Fierce Pharma. (2022). Takeda's narcolepsy drug shows positive results in Phase 3 trials, setting up a potential blockbuster launch. Retrieved from https://www.fiercepharma.com/research/takedas-narcolepsy-drug-shows-positive-results-in-phase-3-trials-setting-up-a-potential-blockbuster-launch
[4] Centessa Pharmaceuticals. (2022). Centessa Pharmaceuticals to advance ORX750 into registrational studies for narcolepsy and idiopathic hypersomnia. Retrieved from https://www.centessapharma.com/news-and-media/press-releases/detail/152/centessa-pharmaceuticals-to-advance-orx750-into-registrational-studies-for-narcolepsy-and-idiopathic-hypersomnia
[5] EvaluatePharma. (2022). World Preview 2022 Outlook: Takeda. Retrieved from https://www.evaluategroup.com/products/world-preview/world-preview-outlook-2022-outlook-takeda/
- The Chinese guidelines for diagnosis and treatment of narcolepsy have recommended Pitolisant (Wakix), a biotech drug, as a first-line treatment therapy, marking a significant shift in pharmaceuticals for managing this rare, chronic disorder.
- Takeda Pharmaceutical Company is developing Oveporexton, an orexin-2 receptor agonist, for the treatment of narcolepsy type 1, indicating a potential advancement in health-and-wellness solutions for patients with this sleep-related medical-condition.
- Centessa Pharmaceuticals is conducting Phase 2a clinical trials for ORX750, an orexin-2 receptor agonist, to address the needs of patients with narcolepsy types 1 and 2, as well as idiopathic hypersomnia, demonstrating a commitment to mental-health issues and chronic-diseases.
- With Takeda's promising drug candidates and Centessa Pharmaceuticals' ongoing clinical trials, in addition to the already approved Pitolisant, the landscape of rare-diseases treatment in China appears to be improving, offering hope for improved management of challenging medical-conditions like narcolepsy.
