Avadel Pharmaceuticals announces final FDA approval of LUMRYZ (sodium oxybate) for extended-release oral suspension as the first and only once-at-bedtime oxybate for cataplexy or excessive daytime sleepiness in adults with narcolepsy. LUMRYZ can now offer people with narcolepsy the opportunity for an uninterrupted night sleep while receiving the full benefit of their prescribed treatment in 1 single bedtime dose that addresses their symptoms of narcolepsy.”ġ. “As we have heard from key stakeholders, previously approved narcolepsy therapies have the potential to disrupt sleep by either causing insomnia or through forced awakening during the middle of the night for their crucial second dose. “Today’s landmark approval and receipt of Orphan Drug Exclusivity represents a major milestone for both Avadel and people living with narcolepsy,” said Greg Divis, chief executive officer of Avadel, in a press release. Lumryz was developed by Avadel Pharmaceuticals. In the study’s phase 3 trial, the drug taken once at bedtime showed statistically significant (p<0.001), clinically meaningful improvement across all primary endpoints in comparison to placebo when evaluated at 6, 7.5, and 9 grams. The FDA based its decision to grant final approval to Lumryz on positive results from the Phase 3 REST-ON clinical study, which was completed in March 2020. Lumryz is an extended-release formulation of sodium oxybate that is now the first and only FDA-approved treatment for this patient population that is indicated to be taken only once at bedtime. Importantly, management also needs to involve sleep hygiene advice, safety measures whenever applicable and guidance with regard to the social sequelae of cataplexy.Ĭataplexy Drop attacks Narcolepsy Pseudocataplexy Sodium oxybate Syncope.The US Food and Drug Administration (FDA) has granted final approval to Lumryz (sodium oxybate) for the treatment of cataplexy and excessive daytime sleepiness in adults who have narcolepsy. Symptomatic treatment is possible with antidepressants and sodium oxybate. Over time, these severe symptoms evolve to the milder adult phenotype, and this pattern is crucial to recognize when assessing the outcome of uncontrolled case series with potential treatments such as immunomodulation. Monoaminergic uptake blocker (5-HT>NE>DA). immediate effects on cataplexy, but abrupt cessation of treatment can induce very severe rebound in cataplexy Clomipramine Tricyclic antidepressant. Cataplexy shows remarkable differences in childhood compared to adults, with profound facial hypotonia and complex active motor phenomena. The treatment of narcolepsy/hypocretin deficiency is well-codified, and involves pharmacotherapies using sodium oxybate, stimu. Currently, the diagnosis of cataplexy is made almost solely on clinical grounds, based on history taking and (home) videos. Childhood narcolepsy, with its profound facial hypotonia, can be confused with neuromuscular disorders, and the active motor phenomenona resemble those found in childhood movement disorders such as Sydenham's chorea. They can be differentiated from cataplexy using thorough history taking, supplemented with (home)video recordings whenever possible. Cataplexy mimics include syncope, epilepsy, hyperekplexia, drop attacks and pseudocataplexy. Next to narcolepsy, cataplexy can sometimes be caused by other diseases, such as Niemann-Pick type C, Prader Willi Syndrome, or lesions in the hypothalamic or pontomedullary region. Moreover, childhood cataplexy differs from the presentation in adults, with a prominent facial involvement, already evident without clear emotional triggers ('cataplectic facies') and 'active' motor phenomena especially of the tongue and perioral muscles. The expression of cataplexy varies widely, from partial episodes affecting only the neck muscles to generalized attacks leading to falls. Although cataplexy is rare, its recognition is important as in most cases, it leads to a diagnosis of narcolepsy, a disorder that still takes a median of 9 years to be diagnosed. This review describes the diagnosis and management of cataplexy: attacks of bilateral loss of muscle tone, triggered by emotions and with preserved consciousness.
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