BRIEF—Breakthrough and fast track designations for pitolisant

USA-based Harmony Biosciences today announced that it has received Breakthrough Therapy and Fast Track designations for its investigational product, pitolisant, from the US Food and Drug Administration.

Harmony acquired rights to pitolisant in October 2017 from France-based Bioprojet which has marketed the product in Europe under the Wakix trade name since its approval by the European Medicines Agency in 2016.

Breakthrough Therapy designation has been granted for pitolisant for the treatment of cataplexy in patients with narcolepsy and is received when a potential new therapy is intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies.

Fast Track designation has been granted for the investigation of pitolisant for the treatment of excessive daytime sleepiness in patients with narcolepsy and the treatment of cataplexy in patients with narcolepsy.

These designations signal the FDA's interest in the development program for pitolisant and provide opportunities for frequent interaction with the agency, the ability to request a rolling new drug application (NDA) submission, as well as the potential for expedited review, with the goal of obtaining approval to market this new medication in the USA in 2019.

Harmony also announced today that the Pitolisant Expanded Access Clinical Evaluation (PEACE) program is open and patients are being enrolled.