The Center for Drug Evaluation (CDE) website in China has indicated that RareStone Group, a China-based rare disease specialist formerly known as Citrine Medicine, has secured priority review status for its in-licensed drug candidate, pitolisant. This status is awarded for the drug’s potential as a treatment for excessive daytime drowsiness (EDS) or cataplexy in adolescents and children over 6 years old with narcolepsy, highlighting the drug’s potential as a pediatric drug as noted by the CDE.
Mechanism of Action and Impact of Pitolisant
Pitolisant is a selective histamine 3 (H3) receptor antagonist/inverse agonist that increases the synthesis and release of histamine, a neurotransmitter that promotes arousal in the brain. By enhancing the activity of histaminergic neurons, pitolisant improves the consciousness and alertness of patients. This mechanism of action makes it a promising treatment for narcolepsy, a condition characterized by EDS and sudden episodes of muscle weakness, or cataplexy.
Regulatory Milestones and Market Access
The drug has already been approved in the EU and US to treat episodic sleeping sickness with or without sudden onset cataplexy, with approvals in March 2016 and August 2019, respectively. Pitolisant obtained breakthrough therapy designation in the US in 2020. RareStone entered into a licensing deal with France-based Bioprojet Pharma in 2020, securing development rights for the drug in China. It was first prescribed in Boao of Hainan province in May 2021. The market filing for pitolisant was accepted for review by the National Medical Products Administration (NMPA) two months later, signaling a potential path to market approval and access for patients in China.-Fineline Info & Tech
