The Breakthrough: Brinsupri (brensocatib)
On August 12, 2025, the U.S. Food and Drug Administration (FDA) approved Brinsupri (brensocatib), a landmark achievement for the bronchiectasis community. This approval marks the first time a therapy has been developed and specifically authorized to treat non-cystic fibrosis bronchiectasis (NCFB), a chronic lung disease causing irreversible airway damage. Previously, care was limited to symptom management and treating infections, but Brinsupri offers a new approach by targeting the disease mechanism itself.
Brinsupri is a neutrophil elastase inhibitor designed to slow the progression of the disease. In bronchiectasis, a vicious cycle of infection and inflammation is driven by the release of proteases, like neutrophil elastase, by neutrophils in the airways. By blocking this enzyme, brensocatib helps to break that cycle. The approval covers adults and pediatric patients aged 12 and older.
Clinical Evidence for Brinsupri
The FDA's decision was based on robust data from clinical trials, including the Phase 3 ASPEN and Phase 2 WILLOW studies. The Phase 3 trial involved over 1,700 participants with NCFB who had experienced at least two antibiotic-treated exacerbations in the previous year.
Key findings demonstrated that patients receiving brensocatib experienced a significantly lower rate of exacerbations compared to those on a placebo. Specifically, the treatment groups had a mean exacerbation rate of 1.0 per year versus 1.3 in the placebo group. The drug also significantly increased the likelihood of patients remaining exacerbation-free over one year (48% vs. 40%). These outcomes underscore Brinsupri's potential to reduce the debilitating flare-ups that characterize the disease.
While Brinsupri primarily focuses on reducing exacerbations, data also showed a slightly less pronounced decline in lung function (FEV1) in the higher-dose group compared to placebo, though this was a small difference. Importantly, Brinsupri's approval signals a new era where therapeutic interventions can target the fundamental pathology of NCFB, rather than just managing its consequences.
Distinguishing Brinsupri from Other Treatments
It is crucial to understand that Brinsupri addresses the chronic inflammatory component of NCFB, which differs from treatments for specific infections that can arise in bronchiectasis patients. A prominent example is Arikayce (amikacin liposome inhalation suspension), which is sometimes confused with a general bronchiectasis treatment. Arikayce, also developed by Insmed, is approved specifically for adults with refractory Mycobacterium avium complex (MAC) lung disease, a type of nontuberculous mycobacterial (NTM) infection that can occur alongside bronchiectasis. While MAC infections are a common complication, Arikayce does not treat the underlying airway damage of bronchiectasis itself. The distinction is critical for understanding treatment goals. The following table compares these two important medications.
| Feature | Brinsupri (brensocatib) | Arikayce (amikacin liposome inhalation) |
|---|---|---|
| Indication | Reduces exacerbations in non-CF bronchiectasis in adults and pediatrics $\ge$ 12. | Treats refractory MAC lung disease in adults with limited or no other options. |
| Mechanism | Inhibits neutrophil elastase, targeting the underlying inflammation. | Liposomal antibiotic, targeting specific MAC bacteria. |
| Administration | Oral capsule, once daily. | Inhaled suspension via a specific nebulizer, once daily. |
| Treatment Target | The chronic inflammatory disease process of NCFB. | A specific bacterial infection (MAC) complicating bronchiectasis. |
| Approval | First drug approved specifically for non-CF bronchiectasis. | First and only approved drug for refractory MAC lung disease. |
Management of Bronchiectasis: A Comprehensive Approach
Pharmacological treatment with Brinsupri is one part of a comprehensive management plan. The full strategy for managing bronchiectasis typically includes a combination of medication, airway clearance, and lifestyle adjustments. Other medications and therapies are used to address different aspects of the disease.
- Antibiotics: Oral, intravenous, or inhaled antibiotics are used to treat acute lung infections (exacerbations). Chronic, intermittent use of inhaled antibiotics (often off-label for bronchiectasis) may be considered for patients with frequent flare-ups, particularly those with chronic P. aeruginosa infection.
- Bronchodilators: Inhaled medications that open the airways and improve breathing, often used before other inhaled treatments.
- Mucus Thinners: Expectorants or hypertonic saline can help loosen sputum, making it easier to clear from the lungs.
Non-pharmacological treatments are also a cornerstone of care:
- Airway Clearance Techniques (ACTs): Essential for helping patients remove the excess mucus that builds up in the airways. Techniques include active cycle breathing, autogenic drainage, and the use of oscillating positive expiratory pressure (PEP) devices or high-frequency chest wall oscillation vests.
- Pulmonary Rehabilitation: Exercise programs and education sessions designed to improve exercise tolerance and overall quality of life.
- Hydration: Staying well-hydrated is important to help keep mucus thin and easier to clear.
- Lifestyle Modifications: Avoiding smoking and air pollutants, maintaining a healthy diet, and receiving vaccinations against influenza and pneumococcal infections are vital.
Conclusion: A New Era for Bronchiectasis Treatment
The approval of Brinsupri represents a monumental turning point for patients living with non-CF bronchiectasis. After decades of relying on treatments that only addressed symptoms or complications, patients now have a targeted therapy to help reduce the frequency of disease exacerbations. This breakthrough, driven by advancements in understanding the inflammatory drivers of the disease, offers a new paradigm for treatment. While Brinsupri is a powerful addition to the medical toolkit, it is still just one part of a comprehensive, personalized care plan that integrates medication, non-pharmacological therapies, and lifestyle management to optimize patient outcomes. For those with comorbid infections like MAC, specific therapies like Arikayce remain a necessary component of treatment.
For more information on the FDA approval of Brinsupri, consult the announcement from NTM Info & Research, which discusses the significance of this event for the bronchiectasis community. NTM Info and Research Inc.
Potential Side Effects of Brinsupri
As with any medication, Brinsupri has potential side effects. Clinical trial data revealed that some patients on brensocatib experienced dermatological and dental issues more frequently than those on placebo. It is important for patients and healthcare providers to monitor for these and other potential adverse effects.
- Cutaneous and Dental Issues: Higher rates of dry skin and periodontitis or gingivitis were noted in clinical trials.
- Infections: Although the drug is designed to reduce inflammation, the possibility of other infections should be monitored.
Patients should always discuss the potential risks and benefits with their healthcare provider.
