ORLANDO, Fla.—Sodium oxybate (Xyrem) demonstrated long-term efficacy in the treatment of narcolepsy in pediatric patients, with a safety profile consistent with that seen in adults. Researchers found no evidence of an increase in depression, anxiety, or suicidality with the medication.
The study results were presented in a pair of posters at Psych Congress 2018.
The drug, a central nervous system depressant, was approved by the US Food and Drug Administration (FDA) on Friday for the treatment of cataplexy and excessive daytime sleepiness in patients aged 7 to 17 years old with narcolepsy. It was approved in 2002 for the treatment of cataplexy in adult patients with narcolepsy.
The study enrolled 106 children, aged 7 to 16, with narcolepsy. Those who were not already taking sodium oxybate were titrated on the medication and entered a stable dose period. A 2-week, double-blind, placebo-controlled randomized-withdrawal period followed. Then, participants entered an open-label evaluation period for up to 47 weeks. In all, the study’s duration was 1 year.
Some 79 participants completed 6 months or more of the study; 46 finished out the year. Sodium oxybate demonstrated effectiveness on cataplexy and excessive daytime sleepiness with little change throughout the study period, researchers reported.
Treatment-emergent adverse events occurring in more than 10% of participants included enuresis, nausea, vomiting, headache, and decreased weight.
Researchers also assessed psychiatric risk linked with long-term use of the drug in children and adolescents. Scores remained in the average range on both the Children’s Depression Inventory 2nd Ed. Self-Report Short Version and the 10-item Multidimensional Anxiety Scale for Children. On the Columbia-Suicide Severity Rating Scale, one participant was identified with suicidal ideation and left the study. Another participant developed acute psychosis but continued in the study after the episode resolved.
Overall, sodium oxybate demonstrated up to a year of efficacy for treating cataplexy and excessive daytime sleepiness in children and adolescents with narcolepsy, with no apparent increased risk of depression, anxiety, or suicidality, researchers concluded.
Jazz Pharmaceuticals, maker of the drug, sponsored the study.
Profant J, Mignot E, Black J, Parvataneni R, Wang YG, Strunc MJ. Sodium oxybate treatment of narcolepsy in pediatric patients: long-term efficacy and safety. Poster presented at Psych Congress 2018: October 25-28, 2018; Orlando, FL. Poster 143.
Profant J, Mignot E, Black J, Parvataneni R, Wang YG, Strunc MJ. Long-term safety and efficacy of sodium oxybate in pediatric patients with narcolepsy: evaluation of depression, anxiety, and suicidal risk. Poster presented at Psych Congress 2018: October 25-28, 2018; Orlando, FL. Poster 319.