FDA Approves First Bronchiectasis Therapy, Brensocatib, for Ages 12 and Up

As of today, August 12, the FDA has approved brensocatib 10 mg and 25 mg for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in adults and children 12 years and older under the name Brinsupri, marking the first disease modifying therapy to be brought to the field.1

"As somebody who's been looking after these patients now for 20 years, I'm desperate to see new drugs for this disease. It can have a devastating effect on patients, and it's very, very difficult when you have limited options for what you can do to help them, and this is going to be a very important option for us," James Chalmers, MBChB, PhD, Professor, Asthma and Lung UK Chair of Respiratory Research, MBChB, PhD, FRCPE, FERS, Clinical Professor (Teaching and Research) of Respiratory Research, Respiratory Medicine and Gastroenterology, University of Dundee, United Kingdom, told HCPLive.

The approval was based off of data from the pivotal, phase 3 ASPEN trial, for which Chalmers served as a primary investigator. The study assessed brensocatib 10 mg, brensocatib 25 mg, or placebo once daily for 52 weeks, followed by 4 weeks off treatment, in 1680 adults and 41 adolescents. It met its primary endpoint of clinically and statistically significantly reducing the annualized rate of pulmonary exacerbations compared with placebo over 52 weeks in both its 10 and 25 mg dosages.2

"This FDA approval represents a potential paradigm shift in how we approach non-cystic fibrosis bronchiectasis," primary investigator Doreen Addrizzo-Harris, MD, the 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 a statement.1 "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." 

In ASPEN, Chalmers and colleagues found that the placebo group had an annualized rate of pulmonary exacerbations of 1.29, the 10-mg brensocatib group had an annualized rate of pulmonary exacerbations of 1.02 (rate ratio, 0.79 (95% CI, 0.69-0.92; adjusted P = .004), and the 25-mg brensocatib group had an annualized rate of pulmonary exacerbations of 1.04 (rate ratio, 0.81 [95% CI, 0.69-0.94; adjusted P = .005]). The hazard ratio for the time to the first exacerbation was 0.81 (95% CI, 0.70-0.95; adjusted P = .02) in the 10-mg group and 0.83 (95% CI, 0.70-0.97; adjusted P = .04) in the 25-mg group.2

Altogether, 48.5% of all patients receiving brensocatib remained exacerbation-free at week 52, compared with 40.3% in the placebo group. Specifically, the 10-mg group had a rate ratio of 1.20 (95% CI, 1.06-1.37; adjusted P = .02) and the 25-mg group had a rate ratio of 1.18 (95% CI, 1.04 to 1.34; adjusted P = .04).2

Benefits were also seen on FEV1, which declined by 50 ml in the 10-mg dose, 24 ml in the 25-mg dose, and 62 ml in the placebo group after 52 weeks. Compared to placebo, the 10-mg group had a least-squares mean (LSM) difference of 11 mL (95% CI, -14 to 37; adjusted P = .38) and the 25-mg group had an LSM difference of 38 mL (95% CI, 11-65; adjusted P = .04).2

Bresocatib has been shown to have a manageable safety profile, with a similar incidence of adverse events across placebo and treatment groups, with the exception of a higher incidence of hyperkeratosis with brensocatib.

"In 2011 the European Respiratory Society… described bronchiectasis as the most neglected disease in respiratory medicine, because there's so many patients, but nobody was really doing research into it, and nobody, nobody was doing any clinical trials. And so, it's really exciting that that's now changing, because our patients really need it to change," Chalmers said.

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. August 12, 2025. https://investor.insmed.com/2025-08-12-FDA-Approves-BRINSUPRI-TM-brensocatib-as-the-First-and-Only-Treatment-for-Non-Cystic-Fibrosis-Bronchiectasis,-a-Serious,-Chronic-Lung-Disease

2. Chalmers JD, Burgel PR, Daley CL, et al. Phase 3 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis. New Eng J Med. 2025; 392 (16): 1569-1581.