Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/C/002616, 2016)
Bioprojet · 2016 · EMA EPAR public assessment report
Report summary
This EMA report details the regulatory assessment of Wakix (pitolisant) for narcolepsy with or without cataplexy. Pitolisant, an H3 receptor antagonist/inverse agonist, demonstrated efficacy in reducing excessive daytime sleepiness (ESS scores) and cataplexy in clinical trials (Harmony I and Ibis), outperforming placebo. Non-clinical studies characterized its pharmacology, pharmacokinetics, and toxicology, noting potential for abuse and QT prolongation at supra-therapeutic doses. While generally well-tolerated, common adverse events included headache and insomnia, with some dose-dependent effects. The report highlights the need for further pharmacokinetic studies and ongoing post-marketing surveillance for long-term safety and specific populations. Quality aspects and the development of 4.5 mg and 18 mg tablets are also covered.
Key findings — cited to the page
| Irritability incidence in pooled clinical trials (Modafinil) | 2.3% | · p.100 |
| Irritability incidence (pitolisant, narcolepsy studies) | 3.2% | · p.74 |
| Irritability incidence (placebo, narcolepsy studies) | 0.6% | · p.74 |
| Irritability incidence in pooled clinical trials (Placebo) | 0.4% | · p.100 |
| Irritability incidence in pooled clinical trials (Pitolisant) | 1.4% | · p.100 |
| Irritability incidence | 1% | · p.97 |
| Irritability incidence (pitolisant, narcolepsy studies pooled) | 3.2% | · p.71 |
| Marketing Authorisation application submission date | 7 May 2014 | Wakix · EU · p.6 |
| Orphan medicinal product designation date | 10 July 2007 | Wakix · EU · p.6 |
| Shelf life of 18 mg strength Wakix | 30 months | Wakix · p.13 |
| Pitolisant tablet strength | 4.5 mg | Wakix · p.9 |
| Prevalence of narcolepsy with cataplexy | 25 and 50 per 100,000 people | · p.8 |
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 →