In a Phase 2 study in pediatric insomnia disorder, in addition to marked improvements in sleep measures, daridorexant 50 mg showed an unexpected pattern of improvement in affect and behavior, cognition, and daily activities in participants with autism spectrum disorder (ASD) or subthreshold ASD characteristics.
Idorsia in-house research data and published scientific research, as well as the unique profile of daridorexant, further support the rationale for its investigation in the treatment of ASD.
Idorsia will therefore pursue a new development program for daridorexant in ASD, with program details expected in the first half of 2027 following discussions with health authorities.
Idorsia Ltd (SIX: IDIA) today announced that it is advancing a new development program to investigate daridorexant in autism spectrum disorder (ASD). The company is working on a dedicated formulation in parallel to discussions with global health authorities about the development program design in ASD, including the doses to be investigated. Further details will be shared in H1 2027.
Roland Wandeler, Chief Executive Officer, commented:
"We are intrigued by the observation from our clinical research that has opened an entirely new scientific avenue for daridorexant and Idorsia. The opportunity to investigate a potential new treatment approach for a condition where treatment options remain extremely limited gives this program particular significance. In addition to its potential impact for people living with ASD and their families, this opportunity demonstrates the value of combining rigorous analysis of our data with deep scientific understanding to uncover important new opportunities for patients and create long-term value for the company.”
The decision is based on findings from Idorsia’s randomized, placebo-controlled Phase 2 study of daridorexant in 165 children and adolescents with insomnia disorder, recently presented at Sleep Europe 2026 (Press release). The study evaluated objectively measured total sleep time after one dose of daridorexant 10, 25, and 50 mg. More than half of participants had diagnosed or subthreshold neurodevelopmental disorder, including ASD or attention-deficit/hyperactivity disorder (ADHD).
Autism spectrum disorder (ASD) is a lifelong neurodevelopmental disorder characterized by persistent impairments in social communication and interaction and by restricted or repetitive behaviors, interests, or activities. Support needs vary widely, with some people requiring lifelong care. ASD affects an estimated 61.8 million people worldwide. In the United States alone, it affects around 1 in 31 eight-year-old children.
Professor Carmen Schröder MD, PhD, Department of Child and Adolescent Psychiatry, Strasbourg University Hospitals, and member of Idorsia’s Autism Advisory Board commented:
"Every child with autism spectrum disorder is different, but for many, difficulties with communication, social interaction, and everyday functioning can shape almost every part of childhood and continue throughout life. Families often carry that burden alongside them, providing extraordinary levels of practical and emotional support while searching for therapeutical interventions that remain extremely limited. With no approved pharmacological treatment addressing the core features of ASD, investigating a new treatment approach offers much-needed hope for children, their caregivers and families, and the clinicians who care for them.”