Japan Approves Dupixent for Pediatric Asthma in Children Aged 6-11 Years
核心洞察
Japan's Ministry of Health, Labour and Welfare has approved Dupixent (dupilumab) for treating bronchial asthma (搜索) in children aged 6 to 11 years with severe or refractory disease inadequately controlled by existing therapy.
The approval is based on the global Phase 3 VOYAGE trial, which demonstrated that Dupixent significantly reduced severe asthma exacerbations by 54% to 65% and improved lung function by 4.68% to 5.32% compared to placebo.
Dupixent becomes the first and only biologic medicine to demonstrate improved lung function in this young patient population in a randomized Phase 3 trial.
Japan's Ministry of Health, Labour and Welfare (MHLW) has granted marketing and manufacturing authorization for Dupixent (dupilumab) for the treatment of bronchial asthma (搜索) in children aged 6 to 11 years with severe or refractory disease whose symptoms are inadequately controlled with existing therapy. This regulatory milestone expands the previous approval in Japan for patients aged 12 years and older, developed jointly by Regeneron Pharmaceuticals and Sanofi.
Clinical Evidence Supporting Approval
The approval is based on robust clinical data from the global Phase 3 VOYAGE trial, which evaluated Dupixent in children aged 6 to 11 years with uncontrolled moderate-to-severe asthma. The study demonstrated significant clinical benefits when Dupixent was added to standard-of-care asthma therapy compared to placebo.
In the VOYAGE trial, Dupixent significantly reduced severe exacerbations by 54% to 65% (p<0.0001) across different patient populations, including the overall population, those with type 2 inflammation (搜索) phenotype (defined by raised blood eosinophils and/or fractional exhaled nitric oxide), and those with raised blood eosinophils. The treatment also improved lung function by 4.68% to 5.32% (p=0.0012, p=0.0009 and p=0.0036, respectively) in these same populations.
Supporting data came from EXCURSION, an open-label extension of VOYAGE that included a sub-study of exclusively Japanese pediatric patients. In this Japanese cohort, Dupixent improved lung function from baseline at 12 weeks and resulted in a low rate of severe asthma exacerbations over one year.
First-in-Class Achievement for Pediatric Population
Dupixent represents a significant therapeutic advancement as the first and only biologic medicine to demonstrate improved lung function in children aged 6 to 11 years in a randomized Phase 3 trial. The drug is a fully human monoclonal antibody that inhibits the signaling of interleukin-4 (搜索) (IL-4 (搜索)) and interleukin-13 (搜索) (IL-13 (搜索)) pathways, which are key and central drivers of type 2 inflammation (搜索).
Safety Profile in Pediatric Patients
The treatment-related adverse events most commonly reported with Dupixent were injection site reactions (erythema, edema and induration) in the VOYAGE trial. In the EXCURSION extension study, the most frequent adverse events included fever, oral herpes, eosinophilia and injection site reactions (erythema and induration).
Addressing Unmet Medical Need
Asthma is one of the most common chronic diseases in children. Despite treatment with current standard-of-care inhaled corticosteroids and bronchodilators, children may continue to experience serious symptoms, such as coughing, wheezing and difficulty breathing. Impaired lung function in young children can have potentially long-lasting impacts, such as reduced lung growth and persistent airway obstruction, if not addressed early.
Patients also may require the use of multiple courses of systemic corticosteroids that carry significant risks. Uncontrolled asthma can interfere with day-to-day activities, like sleeping, attending school and playing sports.
Administration and Dosing
Dupixent is available in Japan as a 200 mg or 300 mg pre-filled syringe or pre-filled pen and is now available for children aged 6 to 11 years with asthma. The medication is intended for subcutaneous injection and is given every two or four weeks based on weight. It can be administered in a clinic or at home by self-administration after training by a healthcare professional. In children younger than 12 years of age, Dupixent should be administered by a caregiver if given at home.
Global Regulatory Status
In addition to asthma, Dupixent is approved in Japan for certain patients with atopic dermatitis (搜索), chronic rhinosinusitis with nasal polyposis (搜索) (CRSwNP), prurigo nodularis (搜索), chronic spontaneous urticaria (搜索) (CSU) and chronic obstructive pulmonary disease (搜索) (COPD). Dupixent has been approved in 50 countries around the world for the treatment of asthma in children aged 6 to 11 years.
Dupixent has received regulatory approvals in more than 60 countries in one or more indications including certain patients with atopic dermatitis (搜索), asthma, CRSwNP, eosinophilic esophagitis (搜索) (EoE), prurigo nodularis (搜索), CSU, COPD and bullous pemphigoid (BP) in different age populations. More than 1,300,000 patients are being treated with Dupixent globally.
Ongoing Development Program
Dupilumab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. To date, dupilumab has been studied across more than 60 clinical trials involving more than 10,000 patients with various chronic diseases driven in part by type 2 inflammation (搜索).
In addition to the currently approved indications, Regeneron and Sanofi are studying dupilumab in a broad range of diseases driven by type 2 inflammation (搜索) or other allergic processes in Phase 3 trials, including chronic pruritus of unknown origin, lichen simplex chronicus and allergic fungal rhinosinusitis.