Pitolisant: First Global Approval

  title={Pitolisant: First Global Approval},
  author={Yahiya Y. Syed},
  • Yahiya Y. Syed
  • Published 20 July 2016
  • Medicine, Biology, Psychology
  • Drugs
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… 

The cost utility of pitolisant as narcolepsy treatment

The objective in this study was to estimate the cost‐effectiveness of pitoliant as monotherapy, and pitolisant as an adjunctive treatment to modafinil, compared with standard treatment.

Evaluation of the abuse potential of pitolisant, a selective H3-receptor antagonist/inverse agonist, for the treatment of adult patients with narcolepsy with or without cataplexy

Pitolisant demonstrated significantly lower potential for abuse compared with phentermine and an overall profile similar to placebo; this suggests a low risk of abuse for pitolisant.

Pitolisant to Treat Excessive Daytime Sleepiness and Cataplexy in Adults with Narcolepsy: Rationale and Clinical Utility

Randomized and observational studies demonstrate pitolisant to be effective in treating both hypersomnolence and cataplexy while generally being well tolerated at prescribed doses.

Pitolisant for treating patients with narcolepsy

Clinical studies showed that pitolisant significantly decreased excessive daytime sleepiness and cataplexy rate versus placebo, and was well tolerated, common adverse reactions were headache, insomnia, nausea, and anxiety.

Update on the treatment of narcolepsy: clinical efficacy of pitolisant

These four trials show that pitolisant is an effective drug to treat EDS and cataplexy in narcolepsy.

Exploring occupancy of the histamine H3 receptor by pitolisant in humans using PET

The objective of the study was to investigate in vivo occupancy of H3 receptors by BF2.649 using PET brain imaging with the H3 receptor antagonist radioligand [11C]GSK189254.

Profile of pitolisant in the management of narcolepsy: design, development, and place in therapy

The review confirmed the effectiveness of pitolisant in treating major clinically relevant narcolepsy symptoms, including cataplexy, as compared to placebo and revealed a safe profile when compared with placebo and active comparators.

Pitolisant in an Adolescent with Prader-Willi Syndrome.

Pitolisant has the potential to improve many symptoms in patients with Prader-Willi syndrome and it appears to be well tolerated with minimal side effects observed, therefore, the use of pitolisant should be considered in patients who fail a psychostimulant trial.



Preclinical evaluation of the abuse potential of Pitolisant, a histamine H3 receptor inverse agonist/antagonist compared with Modafinil

Pitolisant, a histamine H3 receptor inverse agonist/antagonist is currently under Phase III clinical trials for treatment of excessive daytime sleepiness namely in narcoleptic patients. Its drug

Pitolisant versus placebo or modafinil in patients with narcolepsy: a double-blind, randomised trial

Exploratory Phase II Trial to Evaluate the Safety and the Antiepileptic Effect of Pitolisant (BF2.649) in Refractory Partial Seizures, Given as Adjunctive Treatment During 3 Months

There is no evidence for the efficacy of the drug for the regimen that was used, but no firm conclusions can be drawn because the number of included subject was small and the study was not placebo controlled.

Pitolisant, an Inverse Agonist of the Histamine H3 Receptor: An Alternative Stimulant for Narcolepsy-Cataplexy in Teenagers With Refractory Sleepiness

Pitolisant could constitute an acceptable alternative for the treatment of refractory sleepiness in teenagers with narcolepsy and was maintained after a mean of 13 months.

The histamine H3 receptor: from discovery to clinical trials with pitolisant

The cloning of the gene of the H3‐receptor, a member of the superfamily of heptahelical receptors coupled to G proteins, paved the way to the demonstration of the high constitutive activity of the receptor, including its native form, and its participation in the tonic control of histamine release.

Narcolepsy with cataplexy

BF2.649 [1-{3-[3-(4-Chlorophenyl)propoxy]propyl}piperidine, Hydrochloride], a Nonimidazole Inverse Agonist/Antagonist at the Human Histamine H3 Receptor: Preclinical Pharmacology

Preclinical data suggest that BF2.649 is a valuable drug candidate to be developed in wakefulness or memory deficits and other cognitive disorders.