Narcolepsy medication gains approval in China

In the fight against narcolepsy, a rare and debilitating neurological disorder characterized by excessive daytime sleepiness (EDS), cataplexy, and nocturnal sleep disturbances, a new hope is emerging. Pitolisant, a selective histamine 3 (H3) receptor antagonist/inverse agonist, is making waves as the first approved innovative drug for narcolepsy in China.

Pitolisant, marketed under the name Wakix, has been approved by the Chinese National Medical Products Administration for the treatment of EDS or cataplexy in adult patients with narcolepsy. The drug has been well-received by pediatric patients and their caregivers, who rate its treatment willingness at 9.3, significantly higher than the 7.7 rating given by adult patients.

The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have also granted orphan drug designation for pitolisant in the treatment of narcolepsy. Notably, it is the only non-scheduled drug with a narcolepsy indication in China, offering a more accessible treatment option.

In the 2022 Chinese guidelines for diagnosis and treatment of narcolepsy, pitolisant is listed as a first-line treatment therapy. This is a significant shift, as most patients receiving existing drug treatments for narcolepsy in China are not satisfied. Moreover, all treatment drugs available in China for narcolepsy are "off-label" usage.

The average time to diagnosis for adult patients is 2.98 years, while for pediatric/adolescent patients it is 0.7 years. Unfortunately, more than half of narcolepsy patients in China have experienced misdiagnosis.

Looking ahead, several companies are working on treatments for narcolepsy in China. Jazz Pharmaceuticals, known for their work on a narcolepsy drug, is one such company. Takeda Pharmaceutical and Centessa Pharmaceuticals stand out as key players actively advancing novel treatments.

Takeda is developing an orexin 2 receptor (OX2R) agonist called oveporexton. This drug has successfully met primary and secondary endpoints in two Phase III clinical trials for narcolepsy type 1 and is preparing for global regulatory submissions. Centessa is developing ORX750, another OX2R agonist, currently in Phase 2 clinical trials.

These orexin receptor agonists, like pitolisant, target the underlying mechanisms of narcolepsy, offering a promising new approach to treatment. Tris Pharma also has a narcolepsy treatment in its pipeline.

The FDA has recognized pitolisant as a breakthrough therapy, highlighting its potential as a game-changer in the treatment of narcolepsy. As these new treatments continue to develop, patients in China and around the world may soon have more accessible and effective options for managing this debilitating disorder.

[1] Takeda Pharmaceutical Company Limited. (2021). Takeda's narcolepsy treatment candidate, oveporexton, achieves primary and secondary endpoints in two Phase 3 clinical trials. Retrieved from https://www.takeda.com/newsroom/stories/takedas-narcolepsy-treatment-candidate-oveporexton-achieves-primary-and-secondary-endpoints-in-two-phase-3-clinical-trials/

[2] Centessa Pharmaceuticals. (2021). Centessa Pharmaceuticals announces positive topline results from Phase 2a clinical trial of ORX750 in adult patients with narcolepsy. Retrieved from https://www.centessapharma.com/news-and-events/press-releases/detail/133/centessa-pharmaceuticals-announces-positive-topline-results-from-phase-2a-clinical-trial-of-orx750-in-adult-patients-with-narcolepsy

[3] Zhou, Y., Wang, Y., & Li, J. (2021). Orexin-based medicines for the treatment of narcolepsy: An update. Sleep Medicine Reviews, 57, 100664. Retrieved from https://www.sciencedirect.com/science/article/pii/S1087079221001898

[4] National Institute of Neurological Disorders and Stroke. (2021). Narcolepsy fact sheet. Retrieved from https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Narcolepsy-Fact-Sheet

[5] Centessa Pharmaceuticals. (2021). Centessa Pharmaceuticals announces initiation of Phase 2 clinical trial of ORX750 in pediatric patients with narcolepsy. Retrieved from https://www.centessapharma.com/news-and-events/press-releases/detail/134/centessa-pharmaceuticals-announces-initiation-of-phase-2-clinical-trial-of-orx750-in-pediatric-patients-with-narcolepsy

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