FDA Approves WAKIX for Pediatric Cataplexy as Harmony Reports Strong 2025 Results
The FDA has approved WAKIX for cataplexy in pediatric narcolepsy patients, making it the first non-scheduled treatment for all ages. Harmony Biosciences reported 2025 WAKIX revenue of $868.5 million and projects 2026 sales over $1 billion, nearing blockbuster status.
The U.S. Food and Drug Administration has approved WAKIX (pitolisant) for the treatment of cataplexy in pediatric patients 6 years of age and older with narcolepsy, making it the first-and-only FDA-approved non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy. Harmony Biosciences also reported strong 2025 financial results, with WAKIX net product revenue reaching $868.5 million for the full year, and projected 2026 net revenue of $1.0-$1.04 billion on track for blockbuster status.
The FDA's approval of the supplemental new drug application for WAKIX follows a series of regulatory milestones for the drug. WAKIX was first approved by the FDA in August 2019 for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. Approval was expanded to include cataplexy in adult patients in October 2020, and for excessive daytime sleepiness in pediatric patients 6 years and older with narcolepsy in June 2024. WAKIX 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 a selective histamine 3 (H₃) receptor antagonist/inverse agonist and was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.
For the full year ended December 31, 2025, Harmony Biosciences generated $868.5 million in net product revenue for WAKIX, representing approximately 22% year-over-year growth from $714.7 million in 2024. Net product revenue for the fourth quarter of 2025 was $243.8 million, a 21% increase year-over-year. The average number of patients on WAKIX increased by approximately 400 patients to approximately 8,500 patients in Q4 2025. The company projects WAKIX net revenue between $1.0 billion and $1.04 billion for the full year ending December 31, 2026.
In ANDA litigation, the company recently completed settlements with three additional generic filers, bringing the total to six of the seven ANDA filers. The settling parties will receive licenses to launch their generic products no earlier than March 2030 if Harmony is granted pediatric exclusivity, which it is on track to obtain. The company continues to advance efforts toward obtaining pediatric exclusivity for WAKIX, which would add an additional six months of regulatory exclusivity.
The pitolisant franchise is being extended through next-generation formulations. A gastro-resistant formulation (pitolisant GR) has an NDA submission on track for Q2 2026, with an anticipated PDUFA date in Q1 2027. A high-dose formulation (pitolisant HD) has ongoing phase 3 registrational clinical trials in narcolepsy (ONSTRIDE 1) and idiopathic hypersomnia (ONSTRIDE 2), with topline data expected in 2027 and an anticipated PDUFA date in 2028. Utility patents have been filed to extend the franchise into the 2040s.
Harmony Biosciences is also advancing a potential best-in-class orexin-2 agonist (BP1.15205), with a phase 1 trial ongoing and clinical data expected in mid-2026. The company is exploring new pitolisant formulations to pursue broader indications in CNS patient populations where fatigue is a prominent symptom. Harmony has five ongoing phase 3 registrational trials across five distinct CNS indications.