Abstract
Pitolisant (Wakix™) is an inverse agonist of the histamine H3 receptor that is being developed by Bioproject. Oral pitolisant is approved in the EU for the treatment of narcolepsy with or without cataplexy in adults. Pitolisant has received a Temporary Authorization of Use in France for this indication in case of treatment failure, intolerance or contraindication to currently available treatment. Pitolisant has orphan drug designation in the EU and the USA. In the pivotal HARMONY I trial, pitolisant significantly decreased excessive daytime sleepiness versus placebo in adults with narcolepsy with or without cataplexy (primary endpoint). Pitolisant also significantly decreased cataplexy rate versus placebo in these patients. This article summarizes the milestones in the development of pitolisant leading to this first approval for narcolepsy.
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The preparation of this review was not supported by any external funding. 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 author on the basis of scientific completeness and accuracy. Yahiya Syed is a salaried employee of Adis, Springer SBM.
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This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.
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Syed, Y.Y. Pitolisant: First Global Approval. Drugs 76, 1313–1318 (2016). https://doi.org/10.1007/s40265-016-0620-1
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DOI: https://doi.org/10.1007/s40265-016-0620-1