Europe's first Bronchiectasis drug: A disease finally has a treatment
For the first time, the EU has approved a therapy targeting the underlying mechanism of non-CF bronchiectasis — a chronic lung disease that has gone without a treatment for decades, affecting hundreds of thousands of Europeans.
This image is used for illustrative purposes only.
At a Glance
On November 18, 2025, the European Commission granted the first-ever marketing authorization in the EU for a disease-modifying treatment for non-cystic fibrosis bronchiectasis, following a positive opinion from the European Medicines Agency (EMA) on October 16, 2025.
The phase 3 ASPEN clinical trial, published in the New England Journal of Medicine, demonstrated a 19.4% reduction in the annual rate of pulmonary exacerbations at the approved 25 mg dose — both doses tested in the trial showed statistically significant results — across 1,721 patients in 35 countries.
Actual patient access remains contingent on reimbursement decisions made country by country — a separate step from EU authorization, with timelines that vary significantly across member states.
A disease with no treatment — until now
Non-cystic fibrosis bronchiectasis — NCFB — is a chronic lung disease in which the airways become permanently widened as a result of repeated infections and uncontrolled inflammation. Patients live with chronic cough, persistent mucus production, and frequent exacerbations requiring antibiotic courses or hospitalization. Over time, the disease causes irreversible lung tissue damage.
Until 2025, no therapy existed to address this cycle. Management was limited to chest physiotherapy, curative antibiotics, and symptomatic care. Bronchiectasis fell into a specific category of neglect: not rare enough to trigger orphan drug incentives (financial incentives designed to encourage development of therapies for rare diseases), not high-profile enough to attract sustained research investment — a therapeutic blind spot.
The University of Dundee, in Scotland, has been studying neutrophilic inflammation as the driver of this disease for more than fifteen years. It was there that Professor James Chalmers, a consultant respiratory physician, identified the therapeutic pathway of inhibiting DPP1 — an enzyme central to activating the proteases responsible for chronic airway inflammation. The ASPEN trial, which he led, became the largest clinical trial ever conducted in this indication: 1,721 patients, 391 sites, 35 countries.
The mechanism behind the change
Brensocatib (marketed as Brinsupri) is an oral inhibitor of dipeptidyl peptidase 1 (DPP1). By blocking this enzyme, it reduces the activity of neutrophil serine proteases — the agents of inflammation that sustain the cycle of infection and lung destruction. It is the first therapy to act on the disease mechanism itself, rather than its downstream consequences.
What a 19.4% reduction in exacerbations actually represents is not a marginal improvement — it is the difference between no approved therapy and one.
Results from the ASPEN trial, published in the New England Journal of Medicine on April 23, 2025, show that patients taking brensocatib 25 mg — the dose authorized in the EU — had an annual exacerbation rate of 1.04, versus 1.29 in the placebo group, a reduction of 19.4%. The 10 mg dose showed comparable results in the trial but was not included in the EU authorization. The drug also significantly delayed the time to first exacerbation and slowed pulmonary function decline, a benefit rarely demonstrated in this indication. Results were consistent across the trial’s major patient subgroups.
The safety profile proved reassuring. Despite its effect on neutrophil activity, the treatment produced no observed increase in infections or pneumonia — a concern clinicians had raised given neutrophils’ role in immune defense.
Analysis
The mechanism of progress: academic research, multicenter trial, accelerated regulation
Three factors converged to make this approval possible. First, fifteen years of foundational academic research, funded in part by British public grants, yielded a validated therapeutic target. Second, a phase 3 trial conducted across 35 countries produced an evidence base strong enough for accelerated authorization. Third, the European Medicines Agency (EMA), the EU’s drug regulatory body — broadly equivalent in function to the U.S. Food and Drug Administration — activated its accelerated assessment procedure, reserved for medicines addressing a major unmet public health need. This pathway significantly reduced the standard review timeline.
The parallel is telling. The FDA had also granted brensocatib Breakthrough Therapy Designation, its own fast-track mechanism. Both agencies reached the same conclusion within three months of each other, reflecting a convergent global regulatory judgment on clinical priority rather than a European-specific decision.
The documented delta: a break from zero, not an incremental gain
No prior drug had been validated to address the inflammatory mechanism of NCFB. The 19.4% reduction in exacerbations matters less as a percentage than as a category: patients who previously had no approved treatment now have one. Beyond reducing exacerbation frequency, brensocatib also slowed pulmonary function decline — opening what researchers describe as the possibility of modifying the natural course of the disease, rather than managing its episodes.
The citizen impact: hundreds of thousands of patients, uncertain timelines
Brensocatib is approved for patients aged 12 and older who have experienced at least two exacerbations in the prior 12 months — a profile corresponding to the more severe, frequently relapsing forms of the disease. Prevalence estimates vary across studies, but non-CF bronchiectasis is believed to affect hundreds of thousands of patients across Europe.
Effective access, however, is not guaranteed by EU authorization. Each member state negotiates reimbursement conditions separately with the manufacturer. The gap between EU approval and actual patient access — in terms of covered prescriptions — can stretch well beyond a year in some countries, a structural feature of how innovative medicines enter European health systems.
Transferability: a model and its limits
The brensocatib development pathway — academic origin, global multicenter trial, near-simultaneous FDA and EMA approval — offers a replicable model for other chronic respiratory conditions that have fallen below the commercial and research radar. The DPP1 inhibitor drug class is now being explored for other neutrophil-mediated inflammatory diseases, a potential extension of benefit beyond NCFB.
The main constraint is the eligibility profile. The drug targets patients with frequent exacerbations and has not demonstrated efficacy in milder disease forms. The share of NCFB patients who will qualify under the authorization criteria will become clearer as clinical deployment begins.
The deeper question: Europe as regulator and as market
The near-simultaneous approvals — FDA in August 2025, European Commission in November 2025, three months apart — reflect a global regulatory dynamic in which Europe operates as a first-tier co-regulator, not a follower. A positive EMA opinion opens a market of 450 million EU citizens. That weight shapes the economics of drug development, and influences which diseases attract research investment.
This case also raises a harder question. How many chronic conditions, like NCFB before 2025, remain without treatment because the market has never made research sufficiently attractive? The bronchiectasis story is not one of European policy innovation — it is one of academic persistence and clinical rigor eventually clearing regulatory hurdles. The policy question of how to systematically accelerate that process for similarly neglected conditions remains open.
The Bottom Line
The question now is not whether the EU regulatory system worked — it did. The question is whether this case marks the beginning of a shift in how Europe approaches chronically underfunded therapeutic areas, or whether it remains an exception driven by one research team’s fifteen-year commitment. The gap between authorization and reimbursement, country by country, will be the first concrete test. For patients who have managed this disease without any approved therapy, the clock on that process has started.
Sources: European Medicines Agency (EMA) · New England Journal of Medicine (Chalmers et al., 2025) · AJMC · Applied Clinical Trials Online · Respiratory Therapy · Newcastle Hospitals NHS Foundation Trust · University of Dundee