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WAKIX (pitolisant) - Narcolepsy

Opinions on drugs - Posted on Aug 03 2023

Reason for request

Indication extension

Extension d'indication.

 

L'essentiel

Avis favorable au remboursement « chez l’adolescent et l’enfant de plus de 6 ans pour le traitement de la narcolepsie avec ou sans cataplexie ».

 

Quel progrès ?

Un progrès thérapeutique dans la prise en charge.

 

Quelle place dans la stratégie thérapeutique ?

WAKIX (pitolisant) est un traitement de première intention chez l’adolescent et l’enfant de plus de 6 ans pour le traitement de la narcolepsie avec ou sans cataplexie.

Conformément au RCP, l’association du pitolisant avec des médicaments allongeant l’intervalle QT doit être faite sous surveillance étroite.

Le pitolisant peut réduire l’efficacité des contraceptifs hormonaux oraux, il est recommandé d’utiliser une autre méthode de contraception efficace chez la jeune femme en âge de procréer.

 

Recommandations particulières

La Commission recommande que le statut de médicament d’exception soit étendu à cette nouvelle indication. 

Compte-tenu de la complexité de la prise en charge de cette maladie, la décision d’instauration du traitement par WAKIX (pitolisant) devra être prise après proposition documentée issue d’une réunion de concertation thérapeutique avec un centre de référence et de compétences dans la prise en charge de la narcolepsie.


Clinical Benefit
Moderate

The clinical benefit of WAKIX (pitolisant) 4.5 mg and 18 mg film-coated tablet is moderate in the paediatric indication extension to adolescents and children from the age of 6 years for the treatment of narcolepsy with or without cataplexy.

Clinical Added Value
minor

Considering:

  • demonstration of the superiority of pitolisant compared to placebo assessed during a double-blind, randomised study over 8 weeks (including 4 weeks at a fixed dose) in adolescents and children from the age of 6 years for the treatment of narcolepsy with or without cataplexy on:
    • the primary efficacy endpoint of variation in UNS total score, with a modest effect size: mean difference of -3.7 points (CI95% [-6.38; -0.99]; p=0.0073),
    • and on the ranked secondary endpoint of PDSS total score: mean difference of -3.4 points, (CI95% [-5.52; -1.31]; p=0.0015),
  • conflicting results in terms of efficacy on cataplexy during this same study reported in a non-stratified subgroup of patients with type 1 narcolepsy, with a superiority demonstrated versus placebo on the UNS-CTP subscore (mean difference of -1.8 points, CI95% [-3.29; -0.24]; p=0.0229) but not demonstrated on the variation in the number of cataplexy attacks per week,
  • the short-term paediatric safety profile similar to that already known in adults,
  • the medical need only partially met in paediatrics by the recommended therapeutic alternatives, some of which are used off-label,

and despite:

  • questions concerning the relevance of the primary endpoint, corresponding to a scale that has not been validated in children,
  • the lack of quality of life data,
  • the absence of data versus an active comparator in a context in which the majority of patients (64% in the pitolisant group and 69% in the placebo group) had received no prior narcolepsy treatments,

the Committee deems that WAKIX (pitolisant) 4.5 mg and 18 mg provides a minor clinical added value (CAV IV) in the care pathway for the treatment of narcolepsy with or without cataplexy in adolescents and children from the age of 6 years.

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