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Pitolisant Granted Breakthrough Therapy Designation by the FDA

Harmony Biosciences has announced that it has received Breakthrough Therapy and Fast Track designation for pitolisant. The Breakthrough Therapy Designation was created in 2012 to identify drugs that treat a serious or life-threatening disease or condition and demonstrate substantial improvement over existing therapies. FDA expedites the development and review of drugs designated as breakthrough therapies. Typically, such a designation reflects the fact that preliminary clinical evidence demonstrates a clear advantage over available therapies. The advantage can be an effect on irreversible morbidity or mortality, a serious symptom, or a significantly improved safety profile compared to available therapy with evidence of similar efficacy.

The primary intent of the Breakthrough Therapy designation is to help the pharmaceutical company develop, as efficiently as possible, the evidence needed to support drug approval. This may include the FDA working with the pharmaceutical company to design clinical trials to minimize the number of patients exposed to a potentially less efficacious treatment. Thus, with the Breakthrough Therapy designation, Harmony will be able to receive intensive guidance from the review team at the FDA as well as rolling review and priority review. The Breakthrough Therapy designation also brings with it an organizational commitment from senior managers and experienced review staff at the FDA.

"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 in a press release. "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."

Adults with narcolepsy can now access pitolisant via the Pitolisant Expanded Access Clinical Evaluation (PEACE) program ( This program allows physicians in the US to gain clinical experience with the first-in-class investigational product. It also allows patients access to a novel therapeutic option.




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