HRMY Harmony Biosciences

EPX-100 · NCT04462770 registry update

primary_outcomes: ['Percent Change in Countable Motor Seizures Per 28 Days (CMS-28) in the Titration Plus Maintenance Periods Relative to Baseline', 'European Union: Percent Change in Countable Motor Seizures Per 28 Days in the Maintenance Period Relative to Baseline'] → ['Percent Change in Countable Motor Seizures Per 28 Days (CMS-28) in the Titration Plus Maintenance Periods Relative to Baseline', 'European Union: Percent Change in CMS-28 in the Maintenance Period Relative to Baseline']; countries: {'total_sites': 68, 'us_sites': 31} → {'total_sites': 86, 'us_sites': 31}

Harmony Biosciences Reiterates BP-205 MAD Study Data in Q4 2026

The company confirmed it remains on track to report data from the multiple ascending dose study of BP-205 in Q4 2026. No changes to previously guided timing of this or other pipeline milestones.

pitolisant GR · NCT05156047 registry update

primary_outcomes: ['Excessive Daytime Sleepiness'] → ['Change in Epworth Sleepiness Scale (ESS) Score From Week 8 to Week 12']

Harmony Biosciences: Pitolisant GR NDA accepted; PDUFA April 1, 2027

FDA accepted the NDA for pitolisant GR tablets in July 2026. The agency assigned a target PDUFA date of April 1, 2027, setting the regulatory timeline for this gastro-resistant formulation intended to reduce GI side effects and allow therapeutic dosing without titration.

Harmony Biosciences: Pitolisant GR NDA filing 2Q26, PDUFA Q1 2027

The company announced that the NDA for Pitolisant GR (gastro-resistant pitolisant) is on track for submission in Q2 2026. The anticipated PDUFA date is Q1 2027, extending the pitolisant franchise into the 2040s with a formulation designed to reduce GI side effects and allow therapeutic dosing without titration.

Harmony Biosciences to present EPX-100 open-label extension data at AAN 2026

The company will present preliminary open-label extension results from the ongoing Phase 3 ARGUS trial of EPX-100 in Dravet syndrome at the 2026 American Academy of Neurology Annual Meeting. The poster is scheduled for April 22, 2026, 8:00–9:00 AM CT. No new regulatory or clinical outcome data are disclosed in this announcement.

Harmony Biosciences: Pitolisant GR NDA submission Q2 2026, PDUFA Q1 2027

The company stated that the NDA for pitolisant GR will be submitted in Q2 2026 and carries an anticipated PDUFA date in Q1 2027. This sets the next formal regulatory milestone for the gastro-resistant formulation designed to extend the pitolisant franchise into the 2040s.

Harmony Biosciences sets PDUFA for pitolisant GR NDA in Q1 2027

NDA submission for pitolisant GR is guided for Q2 2026 with an anticipated PDUFA date in Q1 2027. The new gastro-resistant formulation is designed to reduce GI side-effects and allow therapeutic dosing without titration, extending the pitolisant franchise into the 2040s.

Harmony Biosciences submits IND for pitolisant HD; Phase 3 trials to start Q4 2025

IND for pitolisant HD submitted to FDA. Phase 3 registrational trials in narcolepsy and idiopathic hypersomnia are scheduled to begin in Q4 2025 with target PDUFA dates in 2028. Utility patents filed with potential exclusivity to 2044.