Idorsia reports positive Phase 2 data for daridorexant in pediatric insomnia, showing dose-dependent sleep improvements, strong safety, and potential beyond sleep regulation.
Written By: Sana Khan BPharm
Reviewed By: Pharmacally Editorial Team
Idorsia has reported positive top-line results from its Phase 2 dose-finding study of daridorexant in pediatric patients with insomnia, advancing its effort to expand the therapy beyond adult populations.
The randomized, placebo-controlled study (NCT05423717) enrolled 165 patients aged 10 to under 18 years, who received daridorexant at doses of 10 mg, 25 mg, or 50 mg, or placebo over two weeks. The trial met its primary endpoint, showing a statistically significant, dose-dependent improvement in objective total sleep time (TST) from baseline as early as Day 1 (p=0.0185), alongside consistent improvements across multiple objective and subjective sleep measures.
Daridorexant demonstrated a safety and tolerability profile comparable to placebo, including at the 50 mg dose. No signals of abuse, withdrawal symptoms, or next-morning residual sleepiness were observed, with data instead indicating improved morning alertness.
The study included children and adolescents both with and without neurodevelopmental disorders such as autism spectrum disorder and attention-deficit/hyperactivity disorder, populations where insomnia is common and treatment options remain limited.
In her remarks, Katharina Lederer, MD, principal investigator, highlighted that pediatric insomnia is often chronic and significantly impacts daytime functioning and development, with an even greater burden in children with neurodevelopmental disorders.
Judith Owens, MD, MPH, Professor Emerita at Harvard Medical School, emphasized the lack of approved pharmacological treatments for pediatric insomnia and the resulting gap in care, particularly in high-risk populations.
From Idorsia, Alberto Gimona, MD, Head of Global Clinical Development & Medical Affairs noted the clear dose-dependent improvements across sleep outcomes and the absence of next-morning residual effects, supporting the therapy’s differentiated profile. Martine Clozel, MD, Pediatrician, CSO and Head of Research, Idorsia added that the findings not only benefit patients but also provide new insights into the role of the orexin system.
Insomnia is a chronic condition characterized by difficulty initiating or maintaining sleep despite adequate opportunity, driven in part by overactive wake signaling and associated with impaired daytime functioning.
Daridorexant works by selectively blocking orexin receptors to regulate wakefulness, offering a targeted alternative to traditional hypnotics. Clinical data published in The Lancet Neurology have previously shown improvements in sleep onset, maintenance, and daytime functioning in adults. Marketed as QUVIVIQ for adult insomnia, daridorexant remains investigational in pediatric patients, with regulatory discussions planned to define next steps.
Beyond improving sleep, the findings suggest that targeting orexin signaling could have wider implications in children with neurodevelopmental disorders, pointing toward a potential new therapeutic avenue extending beyond insomnia itself.
Reference
Idorsia’s daridorexant delivers outstanding results in a Phase 2 study in children with insomnia disorder, 30 March 2026, Idorsia’s daridorexant delivers outstanding results in a Phase 2 study in children with insomnia disorder
Dose-finding Study Assessing the Efficacy, Safety, and Pharmacokinetics of Daridorexant in Subjects Aged 10 to < 18 Years with Insomnia Disorder, ClinicalTrials.gov ID NCT05423717, https://clinicaltrials.gov/study/NCT05423717
About the Writer
Sana Jamil Khan is a B.Pharm graduate with a strong interest in medical writing and scientific communication. Her work focuses on interpreting clinical research, exploring developments in pharmaceutical science, and presenting complex medical information in a clear and accessible manner. She is particularly interested in topics related to human clinical studies, drug safety observations, and emerging therapeutic research.