Harmony Biosciences Holdings, Inc. faced a setback in its sleep drug development as the U.S. Food and Drug Administration (FDA) issued a Refusal to File (RTF) letter for pitolisant, a treatment for excessive daytime sleepiness (EDS) in adult patients with idiopathic hypersomnia (IH). Despite this setback, the company’s net revenue guidance for 2025, ranging from $820 million to $860 million, remains unchanged.
“We are disappointed with this temporary obstacle, but our long-term strategy for pitolisant in treating IH remains steadfast with our focus on Pitolisant HD,” stated Jeffrey M. Dayno, President and CEO of Harmony Biosciences. “We are set to commence a Phase 3 registrational trial of Pitolisant HD in IH in the fourth quarter of 2025, with a projected PDUFA date in 2028.”
While the primary outcome for EDS did not reach statistical significance between pitolisant and placebo in the Phase 3 INTUNE Study, data from the open-label phase indicated significant improvements in patients’ Epworth Sleepiness Scale scores, which were deemed clinically meaningful. Furthermore, most patients in the long-term extension study achieved normal levels of wakefulness and sustained this improvement beyond one year.
Pitolisant, marketed as Wakix in the U.S., is currently approved for EDS or cataplexy in adult narcolepsy patients and EDS in pediatric narcolepsy patients aged 6 and above, but not yet for IH. In the fourth quarter, preliminary net product revenue was reported at approximately $201 million, marking an increase from $168.4 million in the previous year.
Analyst Ami Fadia from Needham maintains a Buy rating for Harmony Biosciences and has revised the price target from $52 to $50. As of the latest report, HRMY stock has dropped by 6.50% to $36.55.
The company’s plans for Pitolisant in treating sleep disorders may have hit a roadblock, but Harmony Biosciences remains committed to advancing its innovative therapies to address the unmet needs of patients.
