The US Food and Drug Administration (FDA) has granted Harmony Biosciences Orphan Drug Designation for pitolisant for the treatment of idiopathic hypersomnia.

Harmony Biosciences is currently evaluating the efficacy and safety of pitolisant in adult patients with idiopathic hypersomnia in a phase 3 registrational INTUNE study, a double-blind, placebo-controlled, randomized withdrawal study. Topline study results are anticipated in the fourth quarter following enrollment completion nine months ahead of plan.

“The FDA’s decision to grant Orphan Drug Designation reinforces our belief in pitolisant as a promising therapy for adult patients with [idiopathic hypersomnia], with the unique added benefit of it being a non-scheduled, once-daily treatment option working through histamine to improve wakefulness,” says Kumar Budur, MD, the company’s chief medical officer. “With the completion of enrollment in our INTUNE study nine months ahead of schedule and topline results expected in Q4, this designation is a significant advance in our clinical and commercial development initiatives.”

Idiopathic hypersomnia is characterized by excessive daytime sleepiness despite sufficient or even long sleep time. Excessive daytime sleepiness in idiopathic hypersomnia is usually not alleviated by naps, longer sleep, or more efficient sleep.

The FDA’s Orphan Drug Designation incentivizes the advancement of promising therapies for rare diseases. Approximately 80,000 people in the US are believed to be affected by idiopathic hypersomnia, with 40,000 currently having been diagnosed.

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