Positive trial results position the next-generation pitolisant formulation for a New Drug Application in early 2026.

Key takeaways:

  • Pitolisant GR met bioequivalence criteria compared with the current 17.8 mg WAKIX tablet.
  • Patients in the dosing optimization study started directly at the therapeutic dose without titration.
  • Safety findings were consistent with existing pitolisant data.
  • Harmony plans an NDA submission in early 2026 with a targeted PDUFA decision in Q1 2027.
  • Patent filings could extend pitolisant GR exclusivity into the mid-2040s.
  • Development of high-dose pitolisant continues to broaden the franchise’s scope in narcolepsy and idiopathic hypersomnia.

Harmony Biosciences Holdings Inc announced positive results from its pivotal bioequivalence study evaluating pitolisant gastro-resistant (GR) formulation. 

With positive findings from both the pivotal bioequivalence and dosing optimization studies, Harmony is on track to submit a New Drug Application (NDA) for pitolisant GR in early 2026. The company is targeting a Prescription Drug User Fee Act date in Q1 2027. With utility patent applications filed for pitolisant GR with potential exclusivity to 2044, Harmony is positioned to extend the pitolisant franchise into the mid-2040s.

“We are very pleased to have demonstrated bioequivalence for pitolisant GR, which, along with the positive findings from our dosing optimization study, confirms its potential as a differentiated next-generation treatment option for narcolepsy patients,” says Kumar Budur, MD, MS, chief medical and scientific officer at Harmony Biosciences, in a release. “The combination of GR coating and ability to initiate treatment at the therapeutic dose without the need for titration, can potentially result in an enhanced overall treatment experience with pitolisant GR.”

The pivotal BE study confirmed that 17.8 mg of pitolisant GR is bioequivalent to existing 17.8 mg WAKIX tablets. The pitolisant GR versus WAKIX AUC and Cmax ratios (%) were 108.46 h*ng/mL (90% CI 103.74 – 113.41) and 99.65 ng/mL (90% CI 91.95 – 108.00), respectively. No new safety or tolerability issues were reported. Importantly, the topline data from the dosing optimization study of pitolisant GR showed 100% of patients successfully initiated treatment at the therapeutic dose of 17.8 mg, eliminating the need for dose titration.

This progress on pitolisant GR underscores the accelerating momentum of the pitolisant franchise, which is further demonstrated by the development of pitolisant HD, a high-dose, enhanced formulation of pitolisant with an optimized pharmacokinetic profile, designed to deliver greater efficacy for excessive daytime sleepiness and other residual symptoms in narcolepsy and idiopathic hypersomnia.

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