19 February 2025 - Harmony Biosciences today announced that it received a refusal to file letter from the US FDA for pitolisant for the treatment of excessive daytime sleepiness in adult patients with idiopathic hypersomnia.

Although the primary outcome for excessive daytime sleepiness between pitolisant and placebo did not reach statistical significance in the randomised withdrawal phase of the Phase 3 INTUNE study, the data from the open-label phase showed that patients experienced improvements on the Epworth Sleepiness Scale that were five times greater than what is recognised as clinically meaningful. Furthermore, the majority of patients in the long-term extension study achieved normal levels of wakefulness and sustained this response beyond one year.

Read Harmony Biosciences press release