RARE Daily

China’s NMPA Approves RareStone’s Wakix to Treat Narcolepsy

July 5, 2023

Rare Daily Staff

The Chinese National Medical Products Administration has approved Rarestone’s Wakix for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy.

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

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.

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 and the U.S. Food and Drug Administration. 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 first recommendation with level A evidence for EDS, cataplexy, hypnagogic hallucinations, and sleep paralysis.

Huge unmet treatment needs exist in China for narcolepsy patients and more than half of the narcolepsy patients in China have experienced misdiagnosis, with an average time to diagnosis of 2.98 years for adult patients and 0.7 years for pediatric/adolescent patients. 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 Shawn Xiang, CEO of RareStone Group.

Photo: Shawn Xiang, CEO of RareStone Group

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