Bioprojet — Wakix EMA EPAR variation assessment report (EMEA/H/C/002616, 2016)
Bioprojet · 2016 · EMA EPAR variation assessment report
Report summary
This report details a Type II variation application for Wakix (pitolisant) to extend its indication for narcolepsy (with or without cataplexy) to children and adolescents aged 6 to less than 18 years. Based on results from the Phase III Study P11-06, pitolisant demonstrated statistically significant improvements over placebo in the Ullanlinna Narcolepsy Scale (UNS) and Paediatric Daytime Sleepiness Scale (PDSS) total scores after 8 weeks. While efficacy was shown for daytime sleepiness and some cataplexy measures, no significant difference was observed for the Weekly Rate of Cataplexy. The safety profile was consistent with adults, with headache and insomnia being common. Pharmacokinetic data from Study P11-11 informed pediatric dosing. The assessment concluded a positive benefit-risk balance, recommending approval of the variation.
Key findings — cited to the page
| Orphan medicinal product designation date | 10 Jul 2007 | Wakix · p.5 |
| Smaller peak of narcolepsy onset | around 36 years of age | · p.7 |
| Wakix authorization year | 2016 | WAKIX · EU · p.9 |
| Orphan medicinal product designation indication | treatment of narcolepsy | Wakix · p.5 |
| Paediatric Investigation Plan (PIP) decision number | P/0057/2021 | · p.5 |
| CHMP opinion date | 26 January 2023 | · p.6 |
| Initial symptoms of narcolepsy occurring before age 18 | over 50% | · p.7 |
| Average prevalence of narcolepsy | approximately 0.025 -0.05% | · p.7 |
| Narcolepsy onset in childhood and adolescence | at least half the cases | · p.7 |
| Long diagnostic delay for narcolepsy | 8–12 years | · p.7 |
| Highest peak of narcolepsy onset | around 15 years of age | · p.7 |
| Narcoleptic patients experiencing first symptoms before age 15 | more than one third | · p.7 |
Source: Bioprojet ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →