XYREM (oxybate de sodium) (narcolepsie pédiatrique)

The Committee deems that the clinical benefit of XYREM (sodium oxybate) is substantial only in the treatment of narcolepsy with cataplexy in drug-resistant adolescents and children from the age of 7 years;

Considering:

• demonstration of the superiority of sodium oxybate compared to placebo assessed during a 2-week double-blind, randomised-withdrawal study in adolescents and children aged from 7 years with narcolepsy with cataplexy:
  • for the primary endpoint of change in median number of weekly cataplexy attacks, with a clinically relevant difference (0 attacks in the sodium oxybate group versus 12.7 in the placebo group; p=0.0002), and
  • for the two ranked secondary clinical endpoints of clinical global impression of change score (CGIc) for the severity of cataplexy attacks and the daytime Epworth Sleepiness Scale (ESS-CHAD),
• the absence of data versus an active comparator in a context in which half of patients (50%) had received prior treatment with stimulants (mainly methylphenidate and modafinil) then concomitantly throughout the study,
• the paediatric safety profile similar to that already known in adults and marked, in particular, by a risk of respiratory depression and depressive disorder, a suicide risk, as well as the risks of abuse or misuse,
• the absence of robust data on quality of life,
• the medical need only partially met by the recommended therapeutic alternatives, some of which are used off-label,

the Committee deems that XYREM (sodium oxybate) provides a minor clinical added value (CAV IV) in the care pathway for the treatment of narcolepsy with cataplexy in drug-resistant adolescents and children from the age of 7 years.