The FDA has approved brensocatib tablets in 10-mg and 25-mg doses as once-daily treatment for non-cystic fibrosis bronchiectasis (NCFB) in adults and children aged 12 years or older, according to a press release from manufacturer Insmed.
NCFB affects approximately 500,000 individuals in the US and millions globally, according to the press release. The condition is distinct from other respiratory infections, in that it involves widening of the airways rather than narrowing.
Exacerbations involving coughing, increased mucus, and shortness of breath are common and disruptive, but targeted treatments have been lacking, according to the company. Brensocatib, marketed as Brinsupri, is a first-in-class inhibitor of DPP-1, designed to prevent the activation of neutrophils that drive the chronic airway inflammation in patients with NCFB, according to the press release.
The approval was based on data from a phase 3 study known as ASPEN and a phase 2 study known as WILLOW. In the ASPEN study, patients with NCFB (1689 adults and 41 adolescents) randomized to 10 mg or 25 mg brensocatib daily had a 21.1% and 19.4% reduction in annual exacerbations, respectively, compared to placebo at 52 weeks. Forced expiratory volume in 1 second declined significantly in brensocatib patients compared to placebo patients, and significantly more brensocatib patients on either dose were exacerbation-free at 52 weeks.
The WILLOW study, a 24-week randomized controlled trial, established the drug’s safety and efficacy based on time to first exacerbation. Brensocatib at both the 10-mg and 25-mg doses significantly extended the time to first exacerbation compared to placebo, and safety data showed no obvious relationships to treatment. Adverse events were mild to moderate in 66%, 88%, and 75% of the placebo, 10 mg, and 25 mg groups, respectively, and serious adverse events were similar across the groups. One death caused by progression of bronchiectasis was reported in a patient in the 25 mg brensocatib group.
The approval authorizes the first treatment that directly targets the root cause of NCFB exacerbations, said ASPEN investigator Doreen Addrizzo-Harris, MD, NYU Grossman School of Medicine, New York City, in the press release. “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,” she said.
Brensocatib is indicated for treatment of NCFB at doses of 10 mg or 25 mg once daily in adults and adolescents aged 12 years or older, with no current contraindications, according to the prescribing information.
Brensocatib is now available through a specialty pharmacy network in the US. In addition, brensocatib applications have been accepted by the European Medicines Agency and the Medicines and Healthcare products Regulatory Agency, and filing of an application in Japan is pending, according to the company, which expects commercial launches in these areas in 2026.
The ASPEN and WILLOW studies were funded by brensocatib manufacturer Insmed.