Jazz Pharmaceuticals Presents Long-Term Safety and Efficacy Data for Xyrem® (sodium oxybate) in Pediatric Patients with Narcolepsy with Cataplexy
"Although the symptoms of narcolepsy typically begin during childhood, there are no cataplexy treatments approved for patients under the age of eighteen," said
In the EXPRESS study, after a two-week double-blind, placebo-controlled withdrawal period (DB), participants entered an open-label safety period for up to 47 weeks, for a total study duration of up to one year. Seventy-nine participants completed ≥6 months, and 46 completed one year. Change in weekly number of cataplexy attacks was calculated from daily cataplexy diaries. EDS was assessed by the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) at each study visit.
The efficacy of sodium oxybate for cataplexy and
The safety profile of sodium oxybate in children and adolescents in this study is similar to that reported in adults, and no new safety concerns were identified following the use of sodium oxybate for up to one year. The most common Treatment-Emergent Adverse Events (TEAEs) (≥5%) were enuresis, nausea, vomiting, headache, decreased weight, decreased appetite, nasopharyngitis and dizziness. Two serious TEAEs occurred (acute psychosis and suicidal ideation), both in sodium oxybate-naïve participants during the open-label titration period. Both events resolved after reducing the dose or discontinuing sodium oxybate, respectively.
Safety assessments included measures of anxiety (Multidimensional Anxiety Scale for Children 10-item [MASC-10]), depressive symptoms (Children's Depression Inventory 2nd Edition Self-Report Short Version [CDI 2:SR(S)]) and suicidality (Columbia-Suicide Severity Rating Scale [C-SSRS]), in addition to TEAEs. T-scores on the MASC-10 were within the average range throughout the study in both sodium oxybate-naïve and on-sodium oxybate participants. T-scores on the CDI 2:SR(S) were within the average range throughout the study in both sodium oxybate-naïve and on-sodium oxybate participants; a slight downward trend was observed in mean CDI 2:SR(S) T-scores over time.
Top-line efficacy results and preliminary safety findings from the EXPRESS study were recently published in The
Xyrem® oral solution, CIII, is indicated for the treatment of cataplexy in narcolepsy and for the treatment of excessive daytime sleepiness (
IMPORTANT SAFETY INFORMATION
Xyrem is a central nervous system (CNS) depressant. In clinical trials at recommended doses obtundation and clinically significant respiratory depression occurred in Xyrem-treated patients. Almost all of the patients who received Xyrem during clinical trials in narcolepsy were receiving central nervous system stimulants.
Xyrem is the sodium salt of gamma hydroxybutyrate (GHB). Abuse of GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death.
Because of the risks of CNS depression, abuse, and misuse, Xyrem is available only through a restricted distribution program called the Xyrem REMS Program, using the central pharmacy that is specially certified. Prescribers and patients must enroll in the program. For further information go to www.XYREMREMS.com or call 1-866-XYREM88® (1-866-997-3688).
Xyrem is contraindicated in combination with sedative hypnotics or alcohol and in patients with succinic semialdehyde dehydrogenase deficiency.
Caution should be used when considering the concurrent use of Xyrem with other CNS depressants. Healthcare providers should caution patients against hazardous activities requiring complete mental alertness or motor coordination within the first 6 hours of dosing or after first initiating treatment until certain that Xyrem does not affect them adversely. The rapid onset of sedation, coupled with the amnestic features of Xyrem, particularly when combined with alcohol, has proven to be dangerous for the voluntary and involuntary user (e.g. assault victim). Patients should be monitored for emergent or increased depression and suicidality and for impaired motor/cognitive function. Episodes of sleepwalking should be fully evaluated and appropriate interventions considered. The amount of daily sodium intake in each dose of Xyrem should be considered in patients sensitive to salt intake. The most common adverse reactions were nausea, dizziness, vomiting, somnolence, enuresis, and tremor.
Please click here to see the full Prescribing Information for Xyrem, including BOXED Warning.
Narcolepsy is a debilitating neurological disorder characterized by excessive sleepiness, and the inability to regulate sleep-wake cycles normally. It affects an estimated one in 2,000 people in the
"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995
This press release contains forward-looking statements, including, but not limited to, statements related to Xyrem as a potential treatment for cataplexy and
1. Dauvilliers Y, Montplaisir J, Molinari N, Carlander B, Ondze B, Besset A, Billiard M. Age at onset of narcolepsy in two large populations of patients in France and Quebec. Neurol 2001; 57:2029-2033
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