PLYMOUTH MEETING, PA — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) announced that the U.S. Food and Drug Administration (FDA) has issued a Refusal to File (RTF) letter for its supplemental New Drug Application (sNDA) for pitolisant. The application sought approval for pitolisant as a treatment for excessive daytime sleepiness (EDS) in adult patients with idiopathic hypersomnia (IH). Despite this setback, Harmony remains focused on its strategic plans for Pitolisant HD and its broader commitment to the sleep/wake and rare neurological disorders space.
“We are disappointed with this short-term setback, but our long-term strategy for pitolisant in IH remains firmly on track with our focus on Pitolisant HD,” said Jeffrey M. Dayno, M.D., President and Chief Executive Officer of Harmony Biosciences. “We made the decision to submit the sNDA based on our belief in pitolisant’s overall benefit-risk profile in IH and our deep commitment to the patient community, despite the challenge associated with this submission.”
Pitolisant HD, an enhanced high-dose formulation of the drug, plays a critical role in Harmony’s future plans. With a provisional patent extending through 2044, Pitolisant HD offers what the company describes as an “optimized profile” and aims to address unmet needs for patients with IH. Harmony plans to initiate a Phase 3 registrational trial for Pitolisant HD in the fourth quarter of 2025, targeting a potential FDA decision in 2028.
“Our planned Phase 3 registrational trial, a double-blind, randomized, parallel-group study in patients with IH, is based on input from the FDA,” explained Kumar Budur, M.D., M.S., Chief Medical and Scientific Officer at Harmony. “We believe this study design will effectively demonstrate the benefit of pitolisant in patients with IH based on the robust clinical response that was observed in the Phase 3 INTUNE Study.”
While the randomized withdrawal phase of the INTUNE Study did not meet its primary statistical endpoint, data from the open-label portion of the trial showed clinically meaningful improvements in patients’ Epworth Sleepiness Scale scores. Long-term results also indicated sustained wakefulness in many participants for over a year.
Harmony’s broader growth strategy remains unchanged, with the company planning to deliver between $820 million and $860 million in net revenue for 2025. The company also emphasized its commitment to WAKIX®, its FDA-approved formulation of pitolisant, which is currently used for the treatment of EDS and cataplexy in adult and pediatric narcolepsy patients.
Looking ahead, Harmony intends to reinforce its market leadership in sleep/wake disorders, leveraging ongoing research and its expanding portfolio. “With the initiation of this pivotal Phase 3 trial in Q4 2025, we will continue to advance pitolisant-based treatments to address [patients’] unmet needs,” said Budur.
Harmony Biosciences states that it remains focused on sustainable growth and ongoing innovation. With plans for the development of Pitolisant HD and its broader pipeline in sleep/wake and rare neurological disorders, the company is positioning itself as a long-term leader in addressing complex conditions and delivering impactful solutions to patients worldwide.
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