Harmony Biosciences receives refusal to file letter from FDA for pitolisant

Harmony Biosciences (HRMY) announced that it received a refusal to file letter from the FDA for pitolisant for the treatment of excessive daytime sleepiness in adult patients with idiopathic hypersomnia. Harmony’s 2025 net revenue guidance of $820M-$860M remains unchanged. Although the primary outcome for EDS between pitolisant and placebo did not reach statistical significance in the randomized 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 recognized 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.