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Pitolisant: Pediatric First Approval

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posted on 2023-05-28, 23:18 authored by Susan Keam


Funding The preparation of this review was not supported by any external funding.

Authorship and Conflict of interest During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Susan J. Keam is a contracted employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability not applicable

Additional information about this Adis Drug Review can be found here.


Pitolisant (WAKIX®), a histamine H3 receptor antagonist/inverse agonist that has been developed by Bioprojet Pharma, is approved in the EU and USA and elsewhere for use in adults with narcolepsy with or without cataplexy. In February 2023, based on clinical data in patients aged 6 to < 18 years, pitolisant received its first approval in adolescents and children from the age of 6 years for the treatment of narcolepsy with or without cataplexy in the EU. This article summarizes the milestones in the development of pitolisant leading to this pediatric first approval for narcolepsy with or without cataplexy. 

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