Respiratory · Clinical trials
Bronchiectasis Clinical Trials
Approved therapies, the pivotal-trial endpoints they were judged on, the magnitude of benefit — and the drugs that failed their endpoints, and why.
Indication overview
About bronchiectasis — and why its trials are hard
Bronchiectasis is a chronic disease defined by permanent, abnormal dilation of the bronchi, driven by a self-perpetuating cycle of impaired mucus clearance, chronic bacterial infection, neutrophil-dominant airway inflammation and progressive structural damage. Patients suffer chronic productive cough, recurrent exacerbations, breathlessness and declining lung function; Pseudomonas aeruginosa colonization marks worse prognosis. Non-cystic-fibrosis bronchiectasis has diverse causes including prior infection, immunodeficiency, primary ciliary dyskinesia and allergic bronchopulmonary aspergillosis. For decades management was entirely supportive and borrowed from other diseases: airway clearance techniques, treatment of exacerbations, long-term macrolides in selected patients, and inhaled antibiotics for chronic Pseudomonas, none FDA-approved specifically for bronchiectasis. Multiple inhaled-antibiotic programs failed to earn a bronchiectasis indication. That changed in August 2025 when brensocatib, an oral reversible inhibitor of dipeptidyl peptidase-1 (DPP-1) that blocks activation of neutrophil serine proteases, became the first drug approved for the condition after the positive Phase 3 ASPEN trial. The annualized pulmonary exacerbation rate is the central efficacy endpoint in this field.
Approved therapies & pivotal evidence
What's been approved — and by how much it moved the endpoint
| Drug (brand) | Approved | Setting | Pivotal trial | Primary endpoint | Magnitude of benefit |
|---|---|---|---|---|---|
| brensocatib (Brinsupri) | 2025 | First and only FDA-approved treatment for non-cystic-fibrosis bronchiectasis, ages 12 and older | ASPEN (Phase 3) | Annualized rate of pulmonary exacerbations over 52 weeks | Significant reduction in annualized exacerbation rate vs placebo (~21% for the 10 mg and ~19% for the 25 mg dose); reduced lung-function decline |
Where trials have failed
Drugs that missed their endpoint — and what contributed
The most instructive lessons in bronchiectasis development come from programmes that failed the endpoint that mattered.
| Drug / trial | Endpoint outcome | What contributed |
|---|---|---|
| inhaled ciprofloxacin dry powder — RESPIRE 1 and RESPIRE 2 (Phase 3) | Inconsistent results across regimens/trials; co-primary exacerbation endpoints not reliably met, and no bronchiectasis approval followed | Heterogeneous patient populations, variable definitions of exacerbation, and dosing-schedule differences produced inconsistent, underpowered signals |
| inhaled liposomal ciprofloxacin — ORBIT-3 and ORBIT-4 (Phase 3) | Only one of the two identical trials met the primary endpoint (time to first exacerbation); overall program failed to secure approval | Lack of replication across the paired trials and inconsistent exacerbation effects undermined the regulatory case |
Choosing the right endpoint
Primary endpoints that matter in bronchiectasis trials
- Annualized pulmonary exacerbation rate — The pivotal efficacy endpoint, used in ASPEN and inhaled-antibiotic trials
- Time to first exacerbation — Co-primary/secondary endpoint in several inhaled-antibiotic programs
- FEV1 / rate of lung-function decline — Physiologic endpoint capturing structural disease progression
- Bacterial load / Pseudomonas density — Microbiologic endpoint relevant to inhaled-antibiotic mechanisms
- Quality of Life-Bronchiectasis (QOL-B) — Validated patient-reported symptom and quality-of-life measure
How iNGENū runs bronchiectasis trials
Physician-led design, built for FDA submission
Endpoint & biomarker strategy
Board-certified specialists design endpoints and patient selection aligned to current FDA guidance for this indication.
FDA-ready data
Built to ICH-GCP and 21 CFR 312.120, with direct FDA submission — data accepted by the FDA, EMA and MHRA.
Faster, lower-cost delivery
~4-week ethics via the TGA CTN scheme, up to 43.5% R&D rebate, and 80–90% below US CRO cost.
Request a fixed-milestone proposal and a tailored endpoint & feasibility summary for this indication.
Frequently asked questions
Bronchiectasis clinical trials — FAQs
Was there any approved bronchiectasis drug before 2025?
How does brensocatib work?
Why did inhaled antibiotics fail to get approved for bronchiectasis?
Ready to discuss your bronchiectasis trial?
Talk to our physician-led team about an FDA-ready, cost-efficient trial design.
Request a proposal