Wakix(pitolisant)
Neurology· 1171 cited findings· 2 source documents
Company: Bioprojet
What the regulatory reviews say
Wakix (pitolisant) is a medication authorized in 2016 for the treatment of narcolepsy. It received orphan medicinal product designation for this indication on July 10, 2007. Narcolepsy onset occurs in childhood and adolescence in at least half of cases, with over 50% of initial symptoms appearing before age 18.
In clinical trials, pitolisant demonstrated efficacy in reducing symptoms of narcolepsy. The estimated LS means difference between pitolisant and placebo for the UNS total score was -3.69 (SE 1.37) [95% CI: -6.38; -0.99], with a p-value of 0.0073. For the PDSS total score, the LS means difference between pitolisant and placebo was -3.41 (SE 1.07) [95% CI: -5.52; -1.31], with a p-value of 0.0015. These results were observed in a double-blind, multicentre, randomized, placebo-controlled trial.
Safety data from an Expanded Access Program showed that 21.4% of patients discontinued due to adverse events. Pitolisant exposure in younger paediatric patients (6 to <12 years) was significantly greater than in older paediatric patients (12 to <18 years), and approximately threefold higher compared to adults. Exposure in older paediatric patients was approximately twofold higher compared to adults.
Key figures — cited to the source page
| UNS total score difference (pitolisant minus placebo) | -3.69 (1.37) [-6.38; -0.99] | Bioprojet — Wakix EMA EPAR variation assessment report (EMEA, p.32 |
| P-value for UNS total score change between pitolisant and placebo | 0.0073 | Bioprojet — Wakix EMA EPAR variation assessment report (EMEA, p.32 |
| PDSS total score difference (pitolisant minus placebo) | -3.41 (1.07) [-5.52; -1.31] | Bioprojet — Wakix EMA EPAR variation assessment report (EMEA, p.34 |
| P-value for PDSS total score difference between pitolisant and placebo | 0.0015 | Bioprojet — Wakix EMA EPAR variation assessment report (EMEA, p.34 |
| Discontinuations due to adverse events | 21.4 % | HARMONY — Wakix FDA Review (NDA211150, ORIG 2, 2020), p.9 |
| Pitolisant exposure in younger paediatric group vs adults | approximately threefold higher | Bioprojet — Wakix EMA EPAR variation assessment report (EMEA, p.10 |
| Orphan medicinal product designation date | 10 Jul 2007 | Bioprojet — Wakix EMA EPAR variation assessment report (EMEA, p.5 |
| Wakix authorization year | 2016 | Bioprojet — Wakix EMA EPAR variation assessment report (EMEA, p.9 |
Source documents
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Figures are as stated in the cited regulatory review documents (different drugs are studied in different trials — values are not head-to-head comparisons). Educational reference only — not medical or investment advice.