November 24, 2025 — Plymouth Meeting, PA — Leads & Copy — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) announced positive results from its pivotal bioequivalence (BE) study evaluating pitolisant gastro-resistant (GR) formulation.
Harmony is on track to submit a New Drug Application (NDA) for pitolisant GR in early 2026, targeting a PDUFA date in Q1 2027, according to the announcement. Positive findings came from both the pivotal BE and dosing optimization studies.
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.
The pivotal BE study confirmed that 17.8mg of pitolisant GR is bioequivalent to existing 17.8mg WAKIX® tablets. The pitolisant GR vs. 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.
The topline data from the dosing optimization study of pitolisant GR showed 100% of patients successfully initiated treatment at the therapeutic dose of 17.8mg, eliminating the need for dose titration.
Harmony Biosciences’ commitment to innovation 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.
With utility patent applications for both pitolisant GR and pitolisant HD filed to potentially extend exclusivity to 2044, Harmony is positioned for sustained market leadership and long-term value creation in the sleep/wake therapeutic space.
WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy. It was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018.
WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.
WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.
In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and at least twice placebo) for WAKIX were insomnia (6%), nausea (6%), and anxiety (5%).
In the placebo-controlled phase of the clinical trial conducted in pediatric patients 6 years and older with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and greater than placebo) for WAKIX were headache (19%) and insomnia (7%).
Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half.
Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required.
Harmony Biosciences is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, Pa.
To report suspected adverse reactions, contact Harmony Biosciences at 1-800-833-7460 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Kumar Budur, MD, MS, Chief Medical and Scientific Officer at Harmony Biosciences
Source: Harmony Biosciences
