Narcolepsy drug gets Chinese approval


RareStone Group, a rare disease-focused company aiming to build China’s first rare disease ecosystem, has announced that the China National Medical Products Administration (NMPA) has approved pitolisant (Wakix) for the treatment of excessive daytime sleepiness (EDS) or cataplexy. in adult patients with narcolepsy.

In China, pitolisant is the first innovative drug approved for narcolepsy and the only non-scheduled drug with an indication for narcolepsy.

Pitolisant (Wakix) is a selective histamine 3 (H3) receptor antagonist/reverse agonist, which was given orphan drug designation for the treatment of narcolepsy by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). It has also been recognized by the FDA as a breakthrough therapy.

Pitolisant has been listed in the Chinese guidelines for the diagnosis and treatment of narcolepsy (2022) as first-line treatment therapy. The Chinese guidelines have emphasized that pitolisant is the first recommendation with level A evidence for EDS, cataplexy, hypnagogic hallucinations, and sleep paralysis.

Narcolepsy is a rare, chronic, debilitating, sleep-wake unstable neurological disorder. Narcolepsy is primarily characterized by excessive daytime sleepiness (EDS), cataplexy, and disturbed sleep at night.

Misdiagnosis of narcolepsy

An unmet need for treatment exists in China for narcolepsy patients. According to the China Narcolepsy Patient Report: A study of disease burden and quality of life, published by CHARD & Peking University, more than half of narcolepsy patients in China are misdiagnosed. Most of the patients had visited several hospitals before receiving a definite diagnosis, with an average diagnosis time of 2.98 years for adult patients and 0.7 years for pediatric/adolescent patients. At the same time, narcolepsy patients generally have strong treatment demands.

On a scale rating the willingness to seek treatment from 0 to 10, the average patient/caregiver score was 9.3, while adult patients were rated 7.7. However, the majority of patients who receive drug treatment are dissatisfied, with great concern about the accessibility of drug therapy. At present, all treatment drugs available in China for narcolepsy are “off-label” use, and most of them are scheduled drugs with strict prescription regulations, seriously hindering patient access to treatment.

“We are excited to present a new therapeutic option for Chinese narcolepsy patients to fill a huge treatment void,” said Shawn Xiang, CEO of RareStone Group.

“We also want to thank our partner, Bioprojet. This fruitful and positive collaboration contributed a lot to bringing this promising treatment drug to narcolepsy patients in China. Aiming to provide more accessible and affordable treatment options for rare disease patients in China, we will continue to work with the patient community, government, industry and stakeholders from all sectors to promote the development and commercialization of innovative treatments for rare diseases.”

Several companies are looking into narcolepsy, although Tris Pharma does have a narcolepsy treatment on the line. Jazz Pharmaceuticals is also working on narcolepsy drugs.


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