The safety and tolerability of pitolisant in the treatment of excessive daytime sleepiness and cataplexy in adult patients with narcolepsy: an open-label, expanded access program in The United States

Bauer, Elizabeth; Davis, Craig W.; Patroneva, Albena; Dayno, Jeffrey M.; Thorpy, Michael J.

doi:10.1016/j.sleep.2019.11.077

Cited by 2 publications

(3 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Interim data are available for 208 participants (59% with NT1), the majority of whom (91%) were titrated to pitolisant 35.6 mg/day; 60% of participants treated with pitolisant were also receiving treatment with one or more concomitant narcolepsy medication (e.g. stimulant, sodium oxybate, modafinil, armodafinil, antidepressant) [22]. Overall, the safety/tolerability profile of pitolisant is consistent with what was found in clinical trials.…”

Section: Safety/tolerabilitysupporting

confidence: 62%

See 1 more Smart Citation

Recently Approved and Upcoming Treatments for Narcolepsy

Thorpy

2020

CNS Drugs

View full text Add to dashboard Cite

Narcolepsy is a chronic, disabling neurologic disorder characterised by excessive daytime sleepiness (EDS) and, in up to 60% of patients, cataplexy. Treatments for narcolepsy are aimed at improving wakefulness (e.g. modafinil, armodafinil, stimulants), reducing cataplexy attacks (e.g. sodium oxybate, venlafaxine), and treating the symptoms of disturbed nocturnal sleep, sleep paralysis and sleep-related hallucinations (e.g. sodium oxybate). In general, medications that increase the release, or inhibit the reuptake, of norepinephrine or dopamine have wake-promoting effects and are useful in managing EDS, whereas medications that inhibit serotonin or norepinephrine reuptake have anticataplectic effects. Modulation of γ-aminobutyric acid B (GABA B) receptors or histamine H 3 receptors (H3Rs) has effects on both EDS and cataplexy. Pitolisant, an H3R antagonist, and solriamfetol, a dopamine and norepinephrine reuptake inhibitor, are the most recently approved treatments for EDS associated with narcolepsy in the European Union (pitolisant) and the USA (pitolisant and solriamfetol). Several new agents are being developed and tested as potential treatments for EDS and cataplexy associated with narcolepsy; these agents include novel oxybate formulations (once-nightly [FT218]; low sodium [JZP-258]), a selective norepinephrine reuptake inhibitor (AXS-12), and a product combining modafinil and an astroglial connexin inhibitor (THN102). This review summarises the mechanisms of action, pharmacokinetics, efficacy, and safety/tolerability of recently approved and emerging treatments for narcolepsy. Key Points Excessive daytime sleepiness and cataplexy are common and disabling symptoms associated with narcolepsy. Emerging treatments, including two recently approved medications (pitolisant and solriamfetol) and several medications still in development (FT218, JZP-258, AXS-12, THN102, SUVN-G3031, TAK-925), provide new options for the treatment of narcolepsy.

show abstract

Section: Safety/tolerabilitysupporting

confidence: 62%

“…In a US-based expanded-access programme, adults with narcolepsy can receive treatment with pitolisant [22].…”

Section: Safety/tolerabilitymentioning

confidence: 99%

Recently Approved and Upcoming Treatments for Narcolepsy

Thorpy

2020

CNS Drugs

View full text Add to dashboard Cite

show abstract

“…The most reported adverse events included headache (8.1%), anxiety (3.8%) and nausea (3.4%). 52 The incidence of adverse effects does appear to be related to dose. A large study involving over 1800 subjects taking pitolisant for a variety of etiologies (including off-label conditions) found that the incidence of adverse events was dose-dependent (11.1% at 4.5 mg/d; 20.3% at 9 mg/d; 32.5% at 18 mg/d; 36.4% at 36 mg/d).…”

Section: Safety and Tolerability Adverse Reactionsmentioning

confidence: 99%

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

Guevarra

Hiensch

Varga

et al. 2020

NSS

View full text Add to dashboard Cite

Narcolepsy is a sleep disorder marked by chronic, debilitating excessive daytime sleepiness and can be associated with cataplexy, sleep paralysis and sleep-related hallucinations. Pharmacological therapy for narcolepsy primarily aims to increase wakefulness and reduce cataplexy attacks. Pitolisant is a first-in-class agent utilizing histamine to improve wakefulness by acting as an antagonist/inverse agonist of the presynaptic histamine 3 receptor. This review summarizes the clinical efficacy, safety and tolerability of pitolisant in treating the symptoms of narcolepsy. Randomized and observational studies demonstrate pitolisant to be effective in treating both hypersomnolence and cataplexy while generally being well tolerated at prescribed doses. The most common adverse reactions include headache, insomnia and nausea.

show abstract

The safety and tolerability of pitolisant in the treatment of excessive daytime sleepiness and cataplexy in adult patients with narcolepsy: an open-label, expanded access program in The United States

Cited by 2 publications

References 0 publications

Recently Approved and Upcoming Treatments for Narcolepsy

Recently Approved and Upcoming Treatments for Narcolepsy

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

Contact Info

Product

Resources

About