RareStone Group, a rare disease-focused company aiming to establish the first rare disease ecosystem in China, announced that on June 30, the Chinese National Medical Products Administration (NMPA)  approved pitolisant (Wakix) for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy. In China, pitolisant is the first approved innovative drug for narcolepsy and the only non-scheduled drug with narcolepsy indication.

Pitolisant (Wakix) is a selective histamine 3 (H3) receptor antagonist/inverse agonist, which was granted orphan drug designation for the treatment of narcolepsy by the European Medicines Agency (EMA) and the U.S. 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 diagnosis and treatment for narcolepsy (2022) as a first-line treatment therapy. The Chinese guidelines have emphasized that pitolisant is the 1^st recommendation with level A evidence for EDS, cataplexy, hypnagogic hallucinations, and sleep paralysis.

Narcolepsy is a rare, chronic, debilitating neurological disorder of sleep-wake state instability. Narcolepsy is primarily characterized by excessive daytime sleepiness (EDS), cataplexy, and nocturnal sleep disturbances.

Huge unmet treatment needs exist in China for narcolepsy patients. According to <China Narcolepsy Patient Report: A study on disease burden and life quality>, published by CHARD & Peking University, more than half of the narcolepsy patients in China have experienced misdiagnosis. Most patients have visited multiple hospitals before receiving a confirmed diagnosis, with an average time to diagnosis 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 treatment willingness rating scale from 0 to 10, the average rating by pediatric patients/caregivers was 9.3, while adult patients rated it at 7.7. However, most patients receiving existing drug treatments are not satisfied, with huge concerns about the accessibility of drug therapy. Currently, all the treatment drugs available in China for narcolepsy are "off-label" usage, and most of them are scheduled drugs with strict prescription regulations, greatly hindering the patient's access to treatment.

"We are pleased to bring a new therapeutic option to Chinese narcolepsy patients to fill in the huge treatment gap," said Dr. Shawn Xiang, CEO of RareStone Group." We would also like to thank our partner, Bioprojet. The fruitful and positive collaboration contributes a lot to bring this promising treatment drug to narcolepsy patients in China. Aiming to bring more accessible and affordable treatment options to rare disease patients in China, we will continue working with the patient communities, government, industry, and stakeholders from all sectors to promote developing and commercializing innovative treatments for rare diseases. "