Regulatory Decision Summary for Nucala

Severe asthma in pediatric patients is a rare, highly complex disorder that is extremely difficult to treat. The burden of disease is high and this population is at risk for near-fatal or fatal exacerbations.

Use of Nucala for the treatment of adolescents (12 to 17 years of age) with severe eosinophilic asthma was established in the pivotal study MENSA (containing 25 adolescents) and supported by study MUSCA (containing 9 adolescents). Overall, adolescents receiving Nucala had a reduction in the rate of clinically significant asthma exacerbations that was consistent with the adult populations in these studies. Nucala was also associated with a clinically meaningful improvement in asthma control.

Evidence supporting the use of Nucala for the treatment of children (6 to 11 years of age) was derived from results in adults and adolescents, as the disease course, pathophysiology, and drug effects (at similar exposure levels) in children are considered sufficiently consistent with adults and adolescents. Additional supporting information were obtained from modelling analysis and an open-label, uncontrolled study in 36 children.

Although a limited number of pediatric patients have been exposed to Nucala, the safety profile in adolescents and children appears to be consistent with that observed in adults from the placebo-controlled severe asthma clinical trials. The most frequently reported treatment-emergent adverse events (AEs) in adolescents and children with severe eosinophilic asthma were headache, injection site reaction, nasopharyngitis, asthma (worsening), bronchitis, and upper respiratory tract infection. The majority of AEs were mild to moderate intensity, and very few were considered to be serious AEs. No pronounced safety risks in the pediatric population were identified from the limited safety information available.

Overall, the Biologic and Radiopharmaceutical Drugs Directorate (BRDD) supports a positive benefit/risk profile for an extension to the indication for Nucala (mepolizumab for injection and mepolizumab injection) for the add-on treatment of adolescents (12 to 17 years of age) and children (6 to 11 years of age) with severe eosinophilic asthma, as well as adding the use of the liquid formulation in pre-filled autoinjector and safety syringe formats for adolescents.