Allschwil, Switzerland, November 4, 2026
Idorsia Ltd. has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to daridorexant for the treatment of insomnia disorder in children and adolescents aged 10 to under 18 years. This regulatory milestone represents an important development in pediatric sleep medicine, an area where there are currently no FDA-approved pharmacological treatments specifically for insomnia disorder. The designation recognizes the preliminary clinical evidence generated through the company’s Phase 2 research and the significant unmet medical need among younger patients experiencing persistent sleep difficulties. It also provides an opportunity for closer collaboration with the FDA during the medicine’s further development. Idorsia aims to advance daridorexant toward a Phase 3 clinical program, with the objective of potentially developing a new treatment option for children and adolescents affected by insomnia.
FDA Grants Breakthrough Therapy Designation to Daridorexant
The FDA’s Breakthrough Therapy Designation is intended to accelerate the development and review of investigational medicines for serious conditions when preliminary clinical evidence indicates the potential for substantial improvement over available treatment options on clinically significant endpoints. The designation provides sponsors with more intensive regulatory guidance, frequent communication with the FDA and support in planning an efficient clinical development pathway. For Idorsia, this designation recognizes the promising findings from its pediatric Phase 2 study of daridorexant, a next-generation dual orexin receptor antagonist (DORA). The medicine works by selectively blocking orexin receptors, which are involved in regulating wakefulness. Unlike traditional sleep medicines that broadly suppress central nervous system activity, daridorexant is designed to reduce excessive wake signaling and support sleep regulation. The medicine was developed with an optimized pharmacokinetic profile intended to provide sleep benefits throughout the night while limiting residual drug exposure the following morning. Daridorexant is already approved for adults with insomnia in certain markets under the brand name QUVIVIQ. However, its use in children and adolescents remains investigational and has not been approved or marketed for this age group in any country.
Phase 2 Study Demonstrates Improvements in Sleep Duration
The Breakthrough Therapy Designation is supported by findings from a multicenter, randomized, double-blind, placebo-controlled Phase 2 clinical study involving 165 children and adolescents aged 10 to under 18 years with insomnia disorder. The trial evaluated three daridorexant dose levels of 10 mg, 25 mg and 50 mg against placebo over a two-week treatment period. The primary objective was to assess the dose-response relationship using changes in total sleep time (TST) measured through polysomnography (PSG), an established method for monitoring sleep patterns and physiological activity during sleep. The analysis demonstrated a statistically significant dose-dependent improvement in total sleep time from baseline on Day 1, with a reported p-value of 0.0185. At the 50 mg dose, participants experienced an increase in sleep duration of approximately 50 minutes overall following a single dose. Among children and adolescents with neurodevelopmental disorders, including participants with autism spectrum disorder (ASD) or attention-deficit/hyperactivity disorder (ADHD), the reported increase was approximately 64 minutes. The study also evaluated sleep quality, morning alertness, daytime functioning, pharmacokinetics, safety and tolerability. According to Idorsia, daridorexant demonstrated a favorable safety and tolerability profile across the evaluated doses, with findings similar to placebo. These results provide preliminary clinical evidence supporting further investigation, although larger and longer-duration studies are required to establish the medicine’s safety and effectiveness in pediatric patients.
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Idorsia Prepares Phase 3 Pediatric Development Program
Following the FDA designation, Idorsia is working toward the next stage of clinical development. The company is consolidating feedback from global health authorities regarding the proposed pediatric Phase 3 study protocol. It expects to initiate the Phase 3 clinical development program in the second half of 2027, subject to further regulatory and development preparations. Pediatric insomnia can affect children’s ability to initiate or maintain sleep and may contribute to difficulties involving attention, learning, emotional regulation, behavior and everyday functioning. The burden can be particularly significant among children with neurodevelopmental conditions, making additional treatment research an important area of unmet medical need. The company is also preparing the full Phase 2 dataset for scientific publication, which is currently under evaluation by a scientific journal. Further clinical investigation will be necessary to confirm the preliminary findings and determine the appropriate benefit-risk profile in younger patients. Overall, FDA Breakthrough Therapy Designation represents an important milestone for Idorsia’s pediatric insomnia research program. With encouraging Phase 2 findings and plans for Phase 3 development, daridorexant could potentially become a new pharmacological treatment option for children and adolescents living with insomnia disorder. Its eventual approval will depend on the successful completion of further clinical studies and regulatory review.
Source: Idorsia press release
