FDA Approves First Targeted Therapy for Non-Cystic Fibrosis Bronchiectasis
Brensocatib offers targeted inflammation control and reduced exacerbations for a serious chronic lung disease affecting hundreds of thousands in the U.S.
Brensocatib (Brinsupri), a first-in-class DPP-1 inhibitor, is now the first FDA-approved treatment for non-cystic fibrosis bronchiectasis, cutting annual exacerbations by up to 21% and potentially setting a new standard of care.
Study Details
Non-cystic fibrosis bronchiectasis is a progressive lung disease affecting about 500,000 people in the U.S. It causes permanent airway widening, mucus buildup, recurrent infections, and inflammation driven largely by overactive neutrophils. Before now, there was no approved therapy to address its underlying inflammatory process. Brensocatib’s approval follows results from the ASPEN phase III trial and the earlier WILLOW phase II trial.
Methodology
ASPEN enrolled adults and adolescents aged 12 and older with a history of exacerbations. Participants were randomized to once-daily brensocatib 10 mg, brensocatib 25 mg, or placebo for one year.
The primary endpoint was the annualized rate of pulmonary exacerbations. Secondary measures included time to first exacerbation, proportion of patients without exacerbations at one year, and lung function decline.
Key Findings
Brensocatib reduced the annualized exacerbation rate by 19–21% vs placebo (P<0.01 for both doses).
48.5% of patients on brensocatib remained exacerbation-free after one year, compared to 40.3% with placebo.
The 25 mg dose significantly slowed lung function decline.
Common side effects included mild upper respiratory infections, gum and skin changes, headache, rash, and hypertension.
Implications for Practice
For the first time, clinicians have an FDA-approved option that directly targets neutrophilic inflammation, a root cause of bronchiectasis exacerbations. Brensocatib’s impact on exacerbation rates and lung function could reduce hospitalizations, antibiotic use, and overall disease burden.
Physicians should counsel patients on potential dermatologic and gum-related side effects and advise against live attenuated vaccines during therapy. This approval may also open the door for earlier, more proactive management of the disease.