Harmony Biosciences Holdings Inc has completed an end-of-phase 2 meeting with the US Food and Drug Administration (FDA) regarding its clinical development plan evaluating pitolisant as a potential treatment for excessive daytime sleepiness in patients ages 6 and older with Prader-Willi syndrome. Harmony plans to initiate a phase 3 registrational study in the fourth quarter of 2023. 

Based on a positive signal observed from the phase 2 proof-of-concept study, Harmony aligned with the FDA on the proposed phase 3 study design elements to support further investigation of pitolisant for children, adolescents, and adults with Prader-Willi syndrome experiencing excessive daytime sleepiness. There is currently no FDA-approved treatment for excessive daytime sleepiness in this patient population.

“We are pleased with the outcome of our end-of-phase 2 meeting with the FDA as we prepare to initiate our phase 3 registrational study, which aims to further investigate the efficacy and safety of pitolisant as a potential treatment for excessive daytime sleepiness in individuals with Prader-Willi syndrome,” says Kumar Budur, MD, chief medical officer at Harmony, in a release. “Building upon the encouraging data obtained from our phase 2 signal detection study, we remain committed to advancing our development program for pitolisant in pursuit of a new indication in people with [Prader-Willi syndrome], given the high unmet medical need in this population.”

There are currently 15,000 to 20,000 people in the United States living with Prader-Willi syndrome, a genetic condition often diagnosed in childhood, according to a release from Harmony, which notes that more than half of these patients experience excessive daytime sleepiness.

Pitolisant is marketed as Wakix in the US and is FDA-approved to treat excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. Pitolisant is not approved for use in patients with Prader-Willi syndrome and is currently being evaluated as an investigational agent in this patient population.

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