FDA Issues Refusal to File for Pitolisant in Idiopathic Hypersomnia Treatment
• Harmony Biosciences receives FDA Refusal to File letter for pitolisant's supplemental New Drug Application in adult idiopathic hypersomnia patients with excessive daytime sleepiness.
• Phase 3 INTUNE study failed to meet primary endpoint for statistical significance, despite showing clinically meaningful improvements in patient wakefulness during open-label phase.
• Company plans to proceed with new Phase 3 registrational trial for Pitolisant HD formulation in Q4 2025, targeting PDUFA date in 2028.
Harmony Biosciences Reports Strong Q3 2024 Results and Pipeline Advancements
• Harmony Biosciences reported $186.0 million in net revenue for Q3 2024, surpassing $2 billion in cumulative net revenue for WAKIX since its launch.
• The company is on track to submit a supplemental New Drug Application (sNDA) for pitolisant in idiopathic hypersomnia (IH) in Q4 2024.
• Harmony's pipeline includes next-generation pitolisant formulations and EPX-100 for rare epilepsies, with Phase 3 trials ongoing for Dravet syndrome and planned for Lennox-Gastaut syndrome.
• Topline data from the ZYN-002 Phase 3 RECONNECT trial in Fragile X syndrome is expected in mid-2025.
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