CHMP recommends EU approval of Brinsupri (brensocatib) for the treatment of non-cystic fibrosis bronchiectasis – Insmed

Insmed announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has adopted a positive opinion recommending approval of its DPP1 inhibitor Brinsupri (brensocatib 25 mg tablets) for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in patients 12 years of age and older with two or more exacerbations in the prior 12 months. If approved, Brinsupri would be first-in-class and the first approved therapy in the European Union to treat NCFB.

The CHMP opinion is based on a comprehensive scientific evaluation of the marketing authorization application, including data from the Phase III ASPEN and Phase II WILLOW studies, which were both published in the New England Journal of Medicine. Brinsupri was reviewed under accelerated assessment by the EMA as it is deemed to be of major interest for public health and therapeutic innovation. Brinsupri had previously been granted Priority Medicines (PRIME) designation by the EMA for the treatment of NCFB following positive Phase II study results from WILLOW. The European Commission (EC) will review the CHMP opinion, with a final decision anticipated in the coming months

ASPEN was a global, randomized, double-blind, placebo-controlled Phase III study to assess the efficacy, safety, and tolerability of brensocatib in patients with non-cystic fibrosis bronchiectasis (NCFB). As part of the ASPEN study’s conduct, more than 460 trial sites were engaged in nearly 40 countries. After excluding sites that did not enroll any patients and all sites in Ukraine, the total number of active sites in ASPEN was 391 sites in 35 countries. Adult patients (ages 18 to 85 years) were randomized 1:1:1 and adolescent patients (ages 12 to <18 years) were randomized 2:2:1 for treatment with brensocatib 10 mg, brensocatib 25 mg, or placebo once daily for 52 weeks, followed by 4 weeks off treatment. The primary efficacy analysis included data from 1,680 adult patients and 41 adolescent patients.

WILLOW was a randomized, double-blind, placebo-controlled, parallel-group, multi-center, multi-national, Phase II study to assess the efficacy, safety and tolerability, and pharmacokinetics of brensocatib administered once daily for 24 weeks in patients with non-cystic fibrosis bronchiectasis (NCFB). WILLOW was conducted at 116 sites and enrolled 256 adult patients diagnosed with NCFB who had at least two documented pulmonary exacerbations in the 12 months prior to screening. Patients were randomized 1:1:1 to receive either 10 mg or 25 mg of brensocatib or matching placebo. The primary efficacy endpoint was the time to first pulmonary exacerbation over the 24-week treatment period in the brensocatib arms compared to the placebo arm.

“With this positive CHMP opinion and the recent FDA approval of BRINSUPRI, we are witnessing the beginning of a new chapter for patients living with NCFB – a serious, chronic lung condition that has long lacked approved treatment options in the European Union,” said Dr. Martina Flammer,  Chief Medical Officer of Insmed. “For too long, patients and families have had to endure the daily burden of this disease without a treatment specifically developed to treat bronchiectasis. At Insmed, our focus remains on delivering first- and best-in-class therapies to patients with serious diseases, and this important milestone brings us one step closer to potentially bringing the first-ever approved treatment to NCFB patients in Europe.”

See citations- Chalmers JD, Haworth CS et al. Phase 2 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis”- J.D. Chalmers and Others. N Engl J Med 2020; 383:2127 •  The causal role of neutrophil serine proteases in the pathogenesis of bronchiectasis was studied with brensocatib, an inhibitor of protease activation. Brensocatib therapy resulted in a longer time to the first bronchiectasis exacerbation than placebo

Chalmers JD, Burgel PR et al. Phase 3 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis.  N Engl J Med 2025; 392:1569  DOI: 10.1056/NEJMoa2411664