In a groundbreaking development, the European Commission has given the green light to BRINSUPRI® (brensocatib) as the inaugural and exclusive treatment for non-cystic fibrosis bronchiectasis (NCFB) in the European Union. This approval is a beacon of hope for those grappling with this severe and progressive lung disease, which can lead to irreversible lung damage and declining lung function.i, iii
NCFB is a unique respiratory condition, distinct from others that cause airway narrowing. Instead, it permanently widens the airways, making it challenging to clear mucus and bacteria, resulting in persistent inflammation and infection.i, iii The disease is characterized by frequent exacerbations, or flares, where symptoms like coughing, increased mucus production, shortness of breath, and fatigue intensify.iv, v
The approval of BRINSUPRI is a significant milestone, offering the first-ever treatment option for NCFB patients aged 12 and above who have experienced two or more exacerbations in the past year. This first-in-class therapy was fast-tracked for review by the EMA due to its potential public health impact.ii
"NCFB significantly impacts daily life, affecting both physical health and emotional well-being," said James Chalmers, MBChB, Ph.D., the ASPEN lead study investigator. "The European Commission's decision is a game-changer, providing a much-needed treatment that can reduce exacerbations, potentially slow disease progression, and transform the treatment landscape for this debilitating condition."
The approval is based on robust scientific evidence, including data from the Phase 3 ASPEN and Phase 2 WILLOW studies published in the New England Journal of Medicine.xii,xiii In ASPEN, patients taking brensocatib 25 mg experienced a 19.4% decrease in annual exacerbation rates compared to placebo.xii Moreover, brensocatib 25 mg met several secondary endpoints related to exacerbations, such as prolonging the time to the first exacerbation and increasing the proportion of patients remaining exacerbation-free during the treatment period.xii Safety was also a key focus, with the most common adverse reactions being headache (9.2%), hyperkeratosis (5.9%), dermatitis (4.2%), rash (4.1%), upper respiratory tract infections (3.9%), and dry skin (3.0%).xii, xiii
"At Insmed, we're committed to bringing new therapies to underserved patient populations," said Martina Flammer, M.D., MBA, Chief Medical Officer. "With brensocatib, we've achieved a significant milestone by providing the first treatment for NCFB in the EU, a population that has historically been overlooked despite their pressing medical needs." And this is where it gets controversial—the accelerated approval process, while expediting access to potentially life-changing treatments, also raises questions about the balance between speed and thoroughness in regulatory reviews.
The EC approval follows a positive opinion from the EMA's Committee for Medicinal Products for Human Use. Insmed is now working with authorities across the EU to ensure eligible patients can access the treatment from early 2026. Applications for brensocatib are also under review in the U.K. and Japan.
NCFB is a complex condition, affecting an estimated 600,000 people in the EU, with potentially two million more undiagnosed.vi, vii, viii, ix, x, xi Insmed is dedicated to raising awareness and providing resources to help patients and healthcare professionals understand and manage this challenging disease.
What are your thoughts on the approval of BRINSUPRI for NCFB? Do you think the accelerated approval process strikes the right balance between speed and safety?
