FDA Grants Breakthrough Therapy, Fast Track Designations to Narcolepsy Treatment

The FDA has granted Harmony Biosciences a Breakthrough Therapy designation for pitolisant, an investigational therapy for the treatment of cataplexy in patients with narcolepsy.

The therapy was also granted a Fast Track designation as well, as there are many unmet needs in the realm of narcolepsy. Misdiagnosis of the condition is common—roughly 25% of those with it have been diagnosed—while it impacts approximately 1 in every 2000 Americans.

“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 Jeffrey Dayno, MD, the chief medical officer at Harmony, said in a statement. “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.”

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