Narcolepsy is a chronic neurological sleep disorder characterized by excessive daytime sleepiness and dysregulation of sleep-wake boundaries, with some patients experiencing cataplexy. It often begins in youth or early adulthood and persists lifelong.
Japan's MHLW granted Orphan Drug Designation to three investigational therapies: gildeuretinol for Stargardt disease, QRX003 for Netherton Syndrome, and E2086 for narcolepsy, each offering potential 10-year market exclusivity.
The FDA approved Lupin's generic pitolisant tablets as bioequivalent to WAKIX, and approved Harmony Biosciences' WAKIX for pediatric cataplexy, making it the first non-controlled treatment for both pediatric and adult narcolepsy patients.
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.
Q1 2026 sleep medicine trends include broader use of AI, expanding obstructive sleep apnea and narcolepsy treatments, and policy changes affecting reimbursement and access.
GSK agreed to license linerixibat to Alfasigma in a deal worth up to $690 million, while Eisai received orphan drug designation in Japan for E2086, a novel selective orexin 2 receptor agonist for narcolepsy.
The FDA approved pitolisant (Wakix) for treating cataplexy in children ages 6 and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
The FDA has expanded approval of Wakix (pitolisant) to treat cataplexy in pediatric patients aged 6 years and older with narcolepsy, making it the first non-controlled substance approved for both pediatric and adult narcolepsy patients with or without cataplexy.
The FDA has approved Harmony Biosciences' Wakix (pitolisant) for treating cataplexy in pediatric patients aged 6 years and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
The FDA has approved pitolisant tablets for treating cataplexy in pediatric patients aged 6 and older with narcolepsy, making it the only non-scheduled therapy approved for both pediatric and adult narcolepsy patients with or without cataplexy.
The FDA approved Harmony Biosciences' supplemental new drug application for WAKIX (pitolisant) to treat cataplexy in pediatric patients 6 years and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07766343 |
Hypersomnia in the Paediatric Population |
NOT_YET_RECRUITING | NA |
| NCT07675135 |
A Phase 3 Efficacy and Safety Study of HBS-301 in Participants With Narcolepsy |
RECRUITING | PHASE3 |
| NCT07625280 |
A Study Evaluating the Efficacy and Safety of Xywav Expanded Dosing vs Placebo in Participants With Narcolepsy or IH |
RECRUITING | PHASE3 |
| NCT07613710 |
A Trial to Test the Safety and Tolerability of Lu AH69593 in Adults That Are Healthy or Have Narcolepsy |
RECRUITING | PHASE1 |
| NCT07540364 |
A Phase 3 Study to Evaluate the Efficacy and Safety of Samelisant in Patients With Narcolepsy (AWAKE) |
NOT_YET_RECRUITING | PHASE3 |
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A Study to Evaluate the Efficacy and Safety of E2086 in Adults With Narcolepsy |
RECRUITING | PHASE2 |
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A Study of HBS-201 (Pitolisant Delayed-release) |
COMPLETED | PHASE1 |
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A Study to Investigate Efficacy, Safety, Tolerability, and Pharmacokinetics of JZP441 Compared With Placebo in Participants With Narcolepsy Type 1 |
TERMINATED | PHASE1 |
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The Impact of Light, Electrical, and Magnetic Neuroregulation Interventions on Sleep-wake Disorders |
RECRUITING | NA |
| NCT06792708 |
Observational Study of LUMRYZ in Narcolepsy |
COMPLETED |
