FDA Grants Harmony Breakthrough Therapy & Fast Track Designations

Harmony Biosciences has received Breakthrough Therapy and Fast Track designations for its investigational product, pitolisant, from the U.S. Food and Drug Administration (FDA). 

These designations signal 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.
"We are very pleased that the FDA has granted pitolisant Breakthrough Therapy and Fast Track designations and believe it reflects the Agency's interest in this investigational product to potentially address an important unmet medical need for patients with narcolepsy," said Harmony's chief medical officer, Jeffrey Dayno, M.D. "We look forward to working with the FDA throughout the submission and review of an NDA for this first-in-class molecule with a novel mechanism of action to treat adult patients with narcolepsy with or without cataplexy."
"Receiving Breakthrough and Fast Track designations for pitolisant from the FDA is welcome news as narcolepsy patients face many challenges," said Harmony's chief executive officer, Bob Repella. "As stated in FDA's Voice of the Patient report on Narcolepsy, it is a debilitating disease that can severely affect a patient's day-to-day functioning and have a devastating impact on their lives."
Harmony also announced that the Pitolisant Expanded Access Clinical Evaluation (PEACE) program is open and patients are being enrolled. This is an open-label Expanded Access Program (EAP) intended to provide treatment with pitolisant to adult patients in the U.S. with excessive daytime sleepiness associated with narcolepsy with or without cataplexy. It is open to all qualified healthcare professionals who wish to participate and interested patients who meet the inclusion/exclusion criteria of the protocol.

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