Bioprojet Pharma: Wakix

Act (FDCA) for Wakix (pitolisant) 4.45 mg and 17.8 mg tablets.

NDA 211150 provides for the use of Wakix (pitolisant) tablets for the following indications which, for administrative purposes, we have designated as follows:

The subject of this action letter is NDA 211150/Original 2. A separate action letter will be issued for NDA 211150/Original 1.

All future submissions to NDA 211150/Original 1 and NDA 211150/Original 2 should specify the NDA number and the Original number to which each submission pertains.

FDA has concluded that your NDA submission does not provide substantial evidence of effectiveness to support the approval of pitolisant for treatment of cataplexy in adult patients with narcolepsy.

Substantial evidence typically consists of at least two adequate and well-controlled trials, each convincing on its own.

2) The subgroup of interest was defined post hoc based on event(s) that occurred postrandomization, which violates the randomization principle and could lead to invalid conclusions.

The Agency has required two positive studies in other narcolepsy development programs (e.g., sodium oxybate, modafinil), as was explained to you during development.

The substantiating study should be a randomized, doubleblind, placebo-controlled, fixed-dose trial of pitolisant for cataplexy that enrolls a meaningful fraction of U.S. patients.

(8) Provide English translations of current approved foreign labeling not previously submitted.