Dupixent Approved by FDA as First Biologic for COPD with Eosinophilic Phenotype
Key Insights
The FDA has approved Dupixent (dupilumab) as an add-on maintenance treatment for adults with inadequately controlled COPD (search) and an eosinophilic phenotype (search).
Approval was based on Phase 3 trials showing significant reductions in COPD (search) exacerbations and improvements in lung function and quality of life compared to placebo.
Dupixent targets the IL-4 and IL-13 (search) pathways, offering a novel approach for COPD (search) patients with type 2 inflammation and elevated blood eosinophils.
The U.S. Food and Drug Administration (FDA) has approved Dupixent (dupilumab) as an add-on maintenance treatment for adults with inadequately controlled chronic obstructive pulmonary disease (COPD (search)) and an eosinophilic phenotype (search). This marks Dupixent as the first biologic medicine approved in the U.S. for these patients, offering a new therapeutic option for a subset of COPD sufferers. The approval was granted to Regeneron and Sanofi, the developers of Dupixent, on September 27, 2024.
This approval addresses a significant unmet need, as approximately 300,000 adults in the U.S. live with inadequately controlled COPD (search) and an eosinophilic phenotype (search). These patients often experience frequent exacerbations despite being on maximal inhaled therapy.
Clinical Trial Data
The FDA's decision was based on data from two pivotal Phase 3 studies, BOREAS (search) and NOTUS (search), which evaluated the efficacy and safety of Dupixent compared to placebo in adults with inadequately controlled COPD (search) and blood eosinophils ≥300 cells per μL. All patients were on maximal standard-of-care inhaled therapy. Key findings from the trials include:
- A 30% and 34% reduction in the annualized rate of moderate or severe COPD exacerbations over 52 weeks in the BOREAS and NOTUS trials, respectively (primary endpoint).
- Numerically greater improvements in post-bronchodilator FEV1 from baseline at week 12 compared to placebo, sustained at 52 weeks (74mL and 68mL in BOREAS and NOTUS, respectively).
- A 51% response in a health-related quality of life measure in both trials compared to 43% and 47% with placebo at 52 weeks, as assessed by a 4-point improvement on the St. George’s Respiratory Questionnaire (SGRQ).
Safety results were consistent with the known safety profile of Dupixent. Common adverse events included viral infection, headache, nasopharyngitis, back pain, diarrhea, arthralgia, and urinary tract infection. Cholecystitis was reported in 0.6% of patients on Dupixent compared to 0.1% on placebo.
Mechanism of Action
Dupixent is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (search) (IL-4) and interleukin-13 (IL-13 (search)) pathways, key drivers of type 2 inflammation. By blocking these pathways, Dupixent helps to reduce inflammation in the lungs and decrease the frequency of COPD (search) exacerbations in patients with an eosinophilic phenotype (search).
Expert Commentary
“This latest FDA approval for Dupixent represents new hope for the hundreds of thousands of COPD (search) patients in the US who can sometimes struggle just to breathe during their everyday lives,” said George D. Yancopoulos, M.D., Ph.D., Board Co-Chair, President and Chief Scientific Officer at Regeneron. “Dupixent has a proven track record as a first-in-class medicine, providing benefit to the many patients suffering from type 2 inflammatory related diseases such as asthma and atopic dermatitis. This latest approval represents an important next chapter for Dupixent, giving those with COPD a novel option that has demonstrated the unprecedented ability to help patients experience fewer exacerbations, while also helping them breathe better and improve quality of life in phase 3 studies.”
Current COPD Treatment Landscape
COPD (search) is a progressive lung disease that causes airflow obstruction, making it difficult to breathe. Symptoms include chronic cough, excessive mucus production, and shortness of breath. Current treatments primarily focus on symptom management through inhaled therapies such as bronchodilators and corticosteroids. However, many patients continue to experience exacerbations, highlighting the need for new treatment options.
Regulatory Context
The FDA evaluated Dupixent under Priority Review. In July 2024, the European Medicines Agency also approved Dupixent for uncontrolled COPD (search) characterized by raised blood eosinophils. Submissions are under review with other regulatory authorities, including in Japan.