Recently Approved and Upcoming Treatments for Narcolepsy

AbstractNarcolepsy 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 (GABAB) receptors or histamine H3 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 ...
Source: CNS Drugs - Category: Neurology Source Type: research

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Sleep disturbances such as excessive daytime sleepiness, central and obstructive sleep apneas, restless legs syndrome, and rapid eye movement sleep dysregulation are prominent in patients with myotonic dystrophy type 1 (DM1). Mild intellectual deficits presented in many patients with DM1. In addition, psychosocial issues caused by neuropsychiatric symptoms are a clinical problem. We herein present the cases of four DM1 patients with sleep disturbances and neuropsychiatric symptoms in the preceding stage of clinically significant muscle symptoms. One of the cases exhibited a sleep disorder and neuropsychiatric symptoms befo...
Source: Frontiers in Neurology - Category: Neurology Source Type: research
Journal of Clinical Sleep Medicine, Ahead of Print.
Source: Journal of Clinical Sleep Medicine : JCSM - Category: Sleep Medicine Authors: Source Type: research
AbstractPurpose of reviewNarcolepsy type 1 (NT1) is a chronic and disabling sleep disorder due to the loss of hypocretinergic neurons in the lateral hypothalamus pathophysiologically linked to an autoimmune process. Current treatment is symptomatic, and no cure is available to date. Immunotherapy is considered a promising future therapeutic option, and this review discusses the rationale for immunotherapy in narcolepsy, current evidences of its effects, outcome measures, and future directions.Recent findingsA limited number of case reports and uncontrolled small case series have reported the effect of different immunothera...
Source: Current Treatment Options in Neurology - Category: Neurology Source Type: research
Publication date: Available online 23 January 2020Source: American Journal of Kidney DiseasesAuthor(s): Jennifer Y. So, Karen M. Warburton, Ilene M. RosenDaytime sleepiness, also known as hypersomnolence, is common among patients receiving maintenance dialysis and following successful kidney transplantation. Sleepiness may be secondary to medical comorbid conditions, medication side effect, insufficient sleep syndrome, and sleep-disordered breathing or the result of a primary central disorder of hypersomnolence, such as narcolepsy. Unrecognized and untreated sleep disorders are associated with substantial morbidity and mor...
Source: American Journal of Kidney Diseases - Category: Urology & Nephrology Source Type: research
The European Commission has approved Sunosi, a Jazz Pharmaceuticals sleep disorder treatment. Sunosi was approved, in 75- and 150-milligram doses, to improve wakefulness and reduce excessive daytime sleepiness in adults with narcolepsy or obstructive sleep apnea who did not respond to primary sleep apnea therapies such as continuous positive airway pressure. Jazz Pharmaceuticals, which is based in Dublin, Ireland, has major operations in Philadelphia where the company has nearly 200 employees.…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
Narcolepsy is a rare brain disorder characterized by excessive daytime sleepiness (EDS), cataplexy, hypnagogic hallucinations, and sleep paralysis. Stimulants have been used to relieve the symptoms of EDS. Narcolepsy symptoms may pose a risk to burn injury...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Burns, Electricity, Explosions, Fire, Scalds Source Type: news
This article aims to review pitolisant.Areas covered: In this paper the chemical properties, mechanism of action, pharmacokinetics, clinical efficacy and safety of pitolisant was introduced, and the development course of drugs for treating narcolepsy is also briefly described. We performed a systematic review of the literature using PubMed and the following keywords were used: "pitolisant" and "narcolepsy", "cataplexy" and "excessive daytime sleepiness" and "histamine 3 receptor".Expert opinion: Pitolisant is a histamine 3 receptor antagonist/inverse agonist. It can activat...
Source: Pharmacological Reviews - Category: Drugs & Pharmacology Authors: Tags: Expert Rev Clin Pharmacol Source Type: research
Abstract Study Objectives: The purpose of this study was to identify patient-centered issues affecting Health-Related Quality of Life (HRQoL) in people with narcolepsy (PWN) and to evaluate patient-reported outcome measures using a mixed-methods approach.Methods: Twenty-nine adults (93% female, mean age = 31 years) with an established diagnosis of narcolepsy (Type I = 58.6%) completed focus group interviews using live videoconferencing. Additionally, participants completed the Patient-Reported Outcomes Measurement Information System (PROMIS) measures along with legacy measures commonly used in ...
Source: Behavioral Sleep Medicine - Category: Sleep Medicine Authors: Tags: Behav Sleep Med Source Type: research
Study Objective:We aimed to evaluate the association between patient-reported outcomes (PROs) and treatment regimen/standardized dose (STD), a measure of drug burden, in patients with narcolepsy type 1 (NT1)/type 2 (NT2) and idiopathic hypersomnia (IH).Methods:Patients age 18 years or older with NT1/NT2 and IH with baseline and≥ 6-month follow-up during 2008–2010 were included. Changes in PROs (Epworth Sleepiness Scale [ESS], Fatigue Severity Scale [FSS], Patient Health Questionnaire 9 [PHQ-9], total sleep time [TST]) by diagnosis, treatment regimen (monotherapy versus polytherapy, sodium oxybate [SO] use), and ST...
Source: Journal of Clinical Sleep Medicine : JCSM - Category: Sleep Medicine Source Type: research
CONCLUSIONS: Due to methodological issues, low sample size, and short duration of the clinical trials as well as high risk of bias for outcome reporting, most of the evidence available for this review is of very low or low quality. For results where quality is low or very low, we are uncertain or very uncertain if the effect estimates are true effects, limiting our conclusions. Specifically, we found that modafinil is no better or worse than placebo at preventing worsening of psychosis; however, we are uncertain about this result. We have more confidence that participants receiving modafinil are no more likely to leave a t...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
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