Breakthrough Therapy Designation granted by the FDA for daridorexant in pediatric insomnia disorder

The designation provides for intensive FDA guidance and closer collaboration on the development of daridorexant in children and adolescents. Daridorexant is a next-generation dual orexin receptor antagonist (DORA) that selectively reduces wake drive rather than broadly inhibiting brain activity. Idorsia designed daridorexant with an optimized pharmacokinetic profile to provide efficacy throughout the night while minimizing residual exposure the following morning. This differentiated profile has been associated with improvements in sleep parameters and daytime functioning in adults with insomnia disorder and may be particularly relevant for children and adolescents.

Pediatric insomnia: a substantial burden on children’s nights and development

Pediatric insomnia is characterized by difficulty initiating or maintaining sleep that is perceived as a problem by the child or/and caregiver due to its severity, chronicity, frequency, and the significant daytime impairment it causes for the child and family. The prevalence (~8-12 %) of the disorder increases from childhood to adolescence. Its burden is particularly high in those with neurodevelopmental disorders, including autism spectrum disorder and attention-deficit/hyperactivity disorder. Insufficient or disrupted sleep can affect attention, learning, emotional regulation, behavior and quality of life, while worsening the symptoms and comorbidities associated with neurodevelopmental disorders. Despite this burden, no pharmacological treatment is currently approved by the FDA for insomnia disorder in pediatric patients.

Breakthrough Therapy Designation recognizes the high unmet need and positive preliminary clinical evidence

Breakthrough Therapy Designation aims to expedite the development and review of drugs intended to treat a serious condition based on preliminary clinical evidence indicating that an investigational medicine may provide a substantial improvement on a clinically significant endpoint, together with the available safety data. It therefore represents important FDA recognition of the clinical results generated with daridorexant to date, and supports the conduct of further studies required to establish its efficacy and safety for potential approval. This closer collaboration is intended to make the development and review of daridorexant as efficient as possible.

Amer Joseph, MD, Chief Medical Officer and Head of Global Clinical Development commented: “This Breakthrough Therapy Designation underscores both the significant unmet medical need faced by children and adolescents living with insomnia disorder and the encouraging clinical evidence generated with daridorexant to date. Daridorexant was specifically designed with an optimized pharmacokinetic and tolerability profile, allowing it to be largely cleared by the next morning and avoiding accumulation with repeated dosing. This profile enabled us to evaluate multiple doses in our pediatric Phase 2 study, including the highest approved adult dose of 50mg. At this dose, daridorexant demonstrated robust efficacy while maintaining a favorable tolerability profile, an especially important consideration in children and adolescents. We look forward to working closely with the FDA as we prepare the Phase 3 program and generate the evidence needed to bring what could become the first FDA-approved pharmacological treatment for pediatric insomnia disorder to patients and their families.”

Next steps in pediatric insomnia

The manuscript describing the full dataset is currently under evaluation with a high-impact scientific journal. In parallel, the team is consolidating supportive comments from global health authorities on the protocol for the pediatric Phase 3 clinical development program. The company expects to initiate the new program in the second half of next year.

Daridorexant for pediatric use is investigational and is not approved or marketed for children or adolescents in any country.

For further information, please contact:

Investor & Media Relations
Idorsia Pharmaceuticals Ltd, Hegenheimermattweg 91, CH-4123 Allschwil
+41 58 844 10 10
investor.relations@idorsia.com / media.relations@idorsia.com
www.idorsia.com