Topline Findings
- FDA approval of Brinsupri (brensocatib) marks the first and only treatment for non-cystic fibrosis bronchiectasis (NCFB) in adults and adolescents 12 years and older, targeting neutrophilic inflammation to address a root cause of exacerbations.
- Phase III ASPEN and Phase II WILLOW trials showed Brinsupri significantly reduced annualized exacerbation rates, prolonged time to first flare, and helped preserve lung function compared with placebo.
- Nearly half of patients on Brinsupri remained exacerbation-free after 52 weeks, with a safety profile comparable to placebo except for a higher incidence of hyperkeratosis.
The FDA has approved Insmed Incorporated’s Brinsupri (brensocatib), marking the first and only therapy indicated for non-cystic fibrosis bronchiectasis (NCFB) in adults and adolescents aged 12 years and older.1
FDA Approves Brinsupri as First Treatment for Non-Cystic Fibrosis Bronchiectasis
- The regulatory action was based on positive results from the Phase III ASPEN (NCT04594369)2 and Phase II WILLOW trials (NCT03218917),3 both of which were published by The New England Journal of Medicine (NEJM).4,5
- In these trials, Brinsupri was found to significantly reduce exacerbation rates, delay time to first flare, and preserve lung function compared to placebo.
“The FDA approval of the first-ever treatment for non-cystic fibrosis bronchiectasis is a historic milestone for patients and for Insmed,” Martina Flammer, MD, MBA, Chief Medical Officer of Insmed, said in a press release. “By keeping patients at the center of everything we do, we have once again delivered a first-in-class medicine for a disease with no prior approved treatments. This is an incredible achievement in medicine.”1
Brinsupri offers first targeted therapy for NCFB patients
- NCFB is a chronic and progressive inflammatory lung disease with a range of clinical manifestations and causes.
- Patients with the disease may carry a significant burden from their daily symptoms, which include chronic cough and sputum.
- These exacerbations have been linked to reduced health-related quality of life, diminished lung function, and higher rates of all-cause mortality.4
“A consistent and established standard of care for bronchiectasis is lacking,” wrote the authors of the ASPEN trial in NEJM. “International guidelines recommend airway clearance techniques, mucoactive agents, and antibiotics for the treatment of infection, but current approaches do not adequately address inflammation or slow disease progression.”4
Pivotal ASPEN and WILLOW trials support approval
- A first-in-class oral, selective, competitive, and reversible dipeptidyl peptidase 1 (DPP1) inhibitor, Brinsupri was developed to inhibit activation of neutrophil enzymes that play a significant role in driving chronic airway inflammation in NCFB.
"Non-cystic fibrosis bronchiectasis deeply affects the lives of people living with this chronic lung condition, impacting both their physical health and emotional well-being," Elisha Malanga, executive director of the Bronchiectasis and NTM Association, said in the release. "Many patients experience frequent flares, which can disrupt daily life and potentially lead to disease progression. The FDA approval of brensocatib represents a significant and long-awaited advancement as the first approved therapy for non-cystic fibrosis bronchiectasis. Our hope is that treatments like this will enable people with bronchiectasis to manage their condition."1
- The double-blind ASPEN trial enrolled 1721 adult and adolescent patients with bronchiectasis, who were randomly assigned in a 1:1:1 ratio for adults and a 2:2:1 ratio for adolescents to receive Brinsupri at doses of 10 mg or 25 mg once daily or placebo.
- The trial’s primary endpoint was annualized rate of adjudicated pulmonary exacerbations over a 52-week period.
Clinical results show Brinsupri reduced exacerbations and preserved lung function
- Among patients in the 10-mg Brinsupri cohort, the annualized rate of pulmonary exacerbations was 1.02 compared to 1.04 in the 25-mg Brinsupri cohort and 1.29 in the placebo cohort (rate ratio, Brinsupri vs. placebo, 0.79 [95% confidence interval {CI}, 0.68 to 0.92; adjusted P=0.004] with the 10-mg dose and 0.81 [95% CI, 0.69 to 0.94; adjusted P=0.005] with the 25-mg dose).
- Across both the 10-mg and 25-mg Brinsupri cohort, 48.5% of patients were exacerbation-free after 52 weeks compared to 40.3% in the placebo cohort.
- In terms of safety, reports of adverse events were similar across the cohorts, aside from a higher incidence of hyperkeratosis in patients treated with Brinsupri.
A potential new standard in non-cystic fibrosis bronchiectasis care
"This FDA approval represents a potential paradigm shift in how we approach non-cystic fibrosis bronchiectasis," ASPEN investigator Doreen Addrizzo-Harris, MD, FCCP, Fiona and Stanley Druckenmiller Professor of Pulmonary, Critical Care and Sleep Medicine at NYU Grossman School of Medicine and director of the NYU Langone Health Bronchiectasis and NTM Program, said in the release. "For the first time, we have a treatment that directly targets neutrophilic inflammation and addresses a root cause of bronchiectasis exacerbations. Based on the strength of the data and the impact we've seen in patients, I believe this could become the new standard in non-cystic fibrosis bronchiectasis care."1
References
1. FDA Approves BRINSUPRI™ (brensocatib) as the First and Only Treatment for Non-Cystic Fibrosis Bronchiectasis, a Serious, Chronic Lung Disease. News release. Insmed Incorporated. August 12, 2025. Accessed August 12, 2025. https://prnmedia.prnewswire.com/news-releases/fda-approves-brinsupri-brensocatib-as-the-first-and-only-treatment-for-non-cystic-fibrosis-bronchiectasis-a-serious-chronic-lung-disease-302527941.html
2. A Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib in Participants With Non-Cystic Fibrosis Bronchiectasis (ASPEN). ClinicalTrials.gov. Updated November 12, 2024. Accessed August 12, 2025. https://clinicaltrials.gov/study/NCT04594369
3. Assessment of INS1007 in Participants With Non-Cystic Fibrosis Bronchiectasis. ClinicalTrials.gov. Updated March 27, 2023. Accessed August 12, 2025. https://clinicaltrials.gov/study/NCT03218917
4. Chalmers J., et al. Phase 3 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis. N Engl J Med 2025;392:1569-1581. DOI: 10.1056/NEJMoa2411664. VOL. 392 NO. 16.
5. Chalmers J., et al. Phase 2 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis. N Engl J Med 2020;383:2127-2137. DOI: 10.1056/NEJMoa2021713. VOL. 383 NO. 22
