Idorsia has reported positive top-line data from its Phase II dose-finding trial evaluating daridorexant, a dual orexin receptor antagonist, for paediatric insomnia disorder.
The randomised study assessed 10mg, 25mg, and 50mg doses in children aged 10 to under 18 with insomnia, to characterise the dose-response relationship of the therapy on objective total sleep time using polysomnography.
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The primary endpoint analysis showed a statistically significant dose-dependent increase in total sleep time from baseline on day one.
In the trial, 165 patients received study medication. It enrolled children with insomnia aged 10 to 11 (21%) and 12 to 17 (79%), including those with neurodevelopmental disorders such as attention-deficit / hyperactivity and autism spectrum disorders.
Safety and tolerability of daridorexant in paediatric patients were confirmed by the study, with no adverse events indicating drug abuse during treatment or signs of withdrawal symptoms after discontinuation reported. Even at the 50mg adult-recommended dose, the safety profile was similar to placebo.
The results also suggest a possible broader role for orexin signalling in children with neurodevelopmental disorders, potentially leading to new therapeutic approaches for this group.
The trial forms part of a paediatric study plan approved by the FDA and a European Union Paediatric Committee (EU PDCO)-endorsed Paediatric Investigation Plan.
Idorsia is planning further discussions with health authorities on the next steps for paediatric insomnia and potential new investigation pathways for children with neurodevelopmental disorders.
Idorsia global clinical development and medical affairs head Alberto Gimona said: “These positive results show for the first time that daridorexant delivers strong, dose-dependent improvements in both objective and subjective sleep outcomes in children.
“While the efficacy is compelling, the safety data are even more striking, including the visual analogue scale where patients and caregivers estimate the feeling of the child’s morning sleepiness – a safety measure of next-morning residual effect – showing no residual sleepiness but improved alertness.”
