Long-term (68 weeks) administration of nemolizumab in paediatric patients aged 6-12 years with atopic dermatitis with moderate-to-severe pruritus: efficacy and safety data from a phase III study

Background A phase III clinical trial in Japanese children aged 6-12 years with atopic dermatitis (AD) and inadequately controlled moderate-to-severe pruritus found that 16 weeks of nemolizumab treatment [30 mg every 4 weeks (Q4W)] was clinically effective and tolerable, with early improvement in pruritus and associated skin signs and a positive impact on patient quality of life (QoL).Objectives To report the findings from the long-term extension period of the study, and evaluate the efficacy and safety profiles of nemolizumab when administered concomitantly with topical agents over 68 weeks.Methods The study included a 16-week randomized placebo-controlled double-blind parallel-group period during which patients received nemolizumab 30 mg or placebo Q4W; those who completed this period could enter a 52-week long-term treatment period during which all patients received nemolizumab Q4W. Efficacy endpoints assessed treatment impact on pruritus, AD signs and QoL. Treatment-emergent adverse events (TEAEs) were tabulated to evaluate long-term safety. The study was registered with the Japan Registry of Clinical Trials (jRCT2080225289).Results Among the 89 paediatric patients evaluated, efficacy outcome measures showed a tendency toward improvement between weeks 16 and 68, and sustained efficacy during the subsequent follow-up period after treatment cessation. At week 68, among patients who received nemolizumab treatment throughout the study, the mean (SD) change from baseline in the 5-level itch score was -1.8 (0.8), the mean (SD) percentage change from baseline in the Average Pruritus Numerical Rating Scale score was -65.9 (25.0), and the mean (SD) percentage change from baseline in the Eczema Area and Severity Index score was -77.1 (23.1). Data from the Dermatitis Family Impact questionnaire indicated that the burden of housework on family members and parent/caregiver fatigue were reduced and the sleep quality of family members was improved by week 16, with further improvement at week 68. The overall safety profile was similar to that recorded in nemolizumab-treated patients aged >= 13 years, with no late-onset TEAEs seen over long-term treatment.Conclusions These data confirm the safety of long-term nemolizumab for paediatric patients with AD, and demonstrate improvements in pruritus, skin symptoms, and patient and caregiver QoL over 68 weeks of treatment. Nemolizumab is a new treatment for atopic dermatitis (or 'AD' for short). The drug is injected under the skin every 4 weeks. It can be used alongside other medicines that are applied to the skin surface. We have already shown that 16 weeks of treatment with nemolizumab improved itch, signs of AD and quality of life in Japanese children with AD who could not control their disease with other medicines. AD can be a lifelong condition and treatments may need to be used for more than 16 weeks.We looked at whether nemolizumab continued to work well and was safe for 68 weeks of treatment. There were 89 children aged 6 to 12 years in the study. We used rating scales and questionnaires to measure the severity of itch, AD eczema and inflammation, and the quality of life of the children and their families. We found that itch and signs of AD reduced over time. The effects continued even after treatment stopped. The children had a better quality of life and their family members reported that they were less tired and had better sleep. Most side effects were mild. We did not see any new side effects after long-term treatment.Based on this study, we can say that long-term treatment with nemolizumab is effective and safe for children with AD and inadequately controlled itch. In Japanese children aged 6-12 years with atopic dermatitis (AD) and inadequately controlled moderate-to-severe pruritus, 16 weeks of nemolizumab treatment (30 mg every 4 weeks) was shown to be clinically effective and tolerable. During a long-term extension period (weeks 16-68), the 5-level itch score, Average Pruritus Numerical Rating Scale and Eczema Area and Severity Index showed a sustained tendency toward improvement, patient quality of life continued to improve, rates of caregiver fatigue decreased and no late-onset treatment-emergent adverse events were seen. These data confirm that long-term (68 weeks) treatment with nemolizumab provides continuing clinical efficacy for paediatric patients with AD and pruritus, with no new treatment concerns.