On World Narcolepsy Day 2021, get a recap of the latest advances in narcolepsy that you might have missed over the last few months, compiled all into one place by the NeurologyLive team.
World Narcolepsy Day 2021, which takes place on September 22, is a day dedicated towards raising awareness of narcolepsy on a global scale. Established by 28 patient advocacy organizations across 6 continents in 2019, World Narcolepsy Day unites the international narcolepsy community to inspire action, increase public knowledge, and elevate the voices of those dealing with narcolepsy worldwide.
It is estimated that anywhere from 135,000 to 200,000 people in the US have narcolepsy; however, since this condition often goes diagnosed, the number may be higher.1 Symptoms of narcolepsy include excessive daytime sleepiness, sleep attacks, cataplexy, sleep paralysis, hallucinations, and disrupted nighttime sleep.
In an effort to continue to raise awareness, NeurologyLive rounded up some of the most notable and recent coverage of news within the field of narcolepsy care. This includes advancements in drug development, the understanding of the disease, and new ways of monitoring its progression.
FT218 (Avadel Pharmaceuticals), an investigational once-nightly sodium oxybate formulation, continued to show its ability as a potential treatment or narcolepsy in the phase 3 REST-ON study (NCT02720744).2 Patients with narcolepsy in the trial demonstrated significant improvements in sleep latency, cataplexy attacks, and on Clinical Global Impression-Improvement scores. The drug is currently under review by the FDA after the agency accepted its new drug application in March, with a Prescription Drug User Fee Act (PDUFA) action date of October 15, 2021.3
After years of only off-label, wake-promoting therapies available for use, the FDA approved the first treatment for idiopathic hypersomnia, JZP-258 (Xywav; Jazz Pharmaceutical). The combination agent of calcium, magnesium, potassium, and sodium oxybates will be available to patients in the fourth quarter of 2021 following Risk Evaluation and Mitigation Strategies implementation.4
A newly developed version of the Narcolepsy Severity Scale, dubbed the pediatric Narcolepsy Severity Scale, was found to be a useful clinical tool associated with self-reported sleepiness, insomnia, and depressive symptoms among school-aged children and adolescents with narcolepsy type 1.5 The easy administration and sensitivity of the tool to detect symptom changes after treatment ensures its future in clinical and research settings.
In the August 2021 issue, Jennifer S. Sun, PhD, discusses the underlying mechanisms of narcolepsy, and why the most important inhibitory neurotransmitter of the mammalian central nervous system is y-aminobutyric acid (GABA), which can promote feelings of happiness and sedation.6 She also discusses y-Hydroxybutyrate, known as GHB or oxybate, which is a physiological compound present in the human body as both a precursor and degradation product of GABA.
Solriamfetol (Sunosi; Jazz Pharmaceuticals), a dopamine/norepinephrine reuptake inhibitor approved to treat excessive daytime sleepiness in adults with narcolepsy or obstructive sleep apnea, continued to show its efficacy among those switching narcolepsy medications.7 The START study consisted of a quantitative retrospective patient chart review among US-based physicians prescribing solriamfetol de novo, in transition from, or in addition to existing medications.
A 3-arm, parallel group, randomized, double-blind, placebo-controlled trial found saffron extract (Affron; Gencor) to have sleep-enhancing effects on adults with unsatisfactory sleep. Additionally, saffron supplementation improved several outcome measures, namely sleep quality ratings, mood ratings after awakening, Insomnia Symptom Questionnaire (ISQ) score, and ISQ-insomnia classifications, when compared to placebo.8