Jazz Pharmaceuticals announced that the Food and Drug Administration (FDA) has approved a supplemental New Drug Application (sNDA) for Xyrem (sodium oxybate) to include an indication for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients with narcolepsy ages ≥7 years old.
Xyrem, a central nervous system (CNS) depressant, was previously only indicated for use in adults. Efficacy in the pediatric population with narcolepsy was demonstrated in the Phase 2/3 EXPRESS study (N=106) which included patients aged 7 to 17 years with narcolepsy with cataplexy. Study patients who were Xyrem-naive at entry underwent open-label titration to reach a tolerable and effective dose. All patients then underwent a 2-week, double-blind, randomized-withdrawal period and were randomized to either remain on Xyrem or receive placebo.
The primary efficacy endpoint was the change in weekly number of cataplexy attacks from baseline to the end of the double-blind period. After this period, patients entered an open-label safety period for up to 47 additional weeks.
Compared with Xyrem-treated patients, those who received placebo experienced a statistically significant increase in weekly cataplexy attacks (median increase 12.7 attacks/week vs 0.3 attacks/week; P <.0001). In addition, patients in the placebo group, during the double-blind treatment period, experienced a statistically significant worsening of EDS vs patients who continued to receive Xyrem (median change in Epworth Sleepiness Scale score from baseline: 3 vs 0; P=.0004).
Regarding safety, the profile was comparable to that reported in adults with no new safety concerns observed with the use of Xyrem for up to 1 year. The most common adverse events reactions in pediatric patients were enuresis, nausea, vomiting, headache, decreased weight, decreased appetite, and dizziness.
Xyrem, a CIII controlled substance, is available as a 0.5g/mL strength oral solution in 180mL bottles. It may only be dispensed to patients enrolled in the Xyrem REMS program.
For more information call (800) 520-5568 or visit Xyrem.com.