Pitolisant(Wakix)

Neurology· 572 cited findings· 1 source document

Company: Bioprojet

What the regulatory reviews say

Pitolisant (Wakix) is a medication designated as an orphan medicinal product on 10 July 2007 (EU/3/07/459). A Marketing Authorisation application was submitted on 7 May 2014, leading to a CHMP positive opinion on 19 November 2015. The drug is indicated for the treatment of narcolepsy.

In efficacy trials, pitolisant demonstrated a mean ESS score reduction from baseline of -4.6, compared to -3.6 for placebo and -7.8 for modafinil. The ESS score decrease for pitolisant versus placebo was -1.94, with a responder rate of 0.60 (p-value 0.008). Pitolisant also showed an increase in MWT of +1.14 minutes, with a p-value of 0.009 versus placebo. The cataplexy rate for pitolisant versus modafinil was 0.70. The non-inferiority margin for pitolisant versus modafinil was -2 points, with a difference of -2.75.

Regarding safety, premature withdrawals were observed in 7 patients receiving pitolisant, 3 for modafinil, and 2 for placebo. Four premature withdrawals in the pitolisant group were due to adverse events. Pitolisant showed a QTc interval prolongation of +10% at a dose of 1.5 mg/kg in telemetered dogs. The elimination half-life (t½) of pitolisant is 7*24 hours. A final report for a Post-Authorisation Safety Study is due in Q3 2023.

Key figures — cited to the source page

Mean ESS score reduction from baseline (pitolisant)	-4.6	Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/, p.54
Mean ESS score reduction from baseline (modafinil)	-7.8	Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/, p.54
ESS score decrease (pitolisant vs placebo)	-1.94	Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/, p.55
Responder rate (pitolisant vs placebo)	0.60	Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/, p.55
MWT increase (pitolisant)	+1.14	Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/, p.55
Cataplexy rate (pitolisant vs modafinil)	0.70	Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/, p.59
Premature withdrawals (pitolisant)	7	Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/, p.54
Orphan medicinal product designation number	EU/3/07/459	Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/, p.6

Source documents

Bioprojet — Wakix EMA EPAR public assessment report (EMEA/H/C/002616, 2016) (2016)

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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.