Brensocatib’s Benefits Extend to Lung Structure – Medscape

Report on Brensocatib’s Impact on Non-Cystic Fibrosis Bronchiectasis and its Alignment with Sustainable Development Goals

Introduction: Advancing Global Health Targets

A new therapeutic agent, brensocatib, has received FDA approval for the treatment of non-cystic fibrosis bronchiectasis (NCFB). Clinical trial data indicates the drug not only manages symptoms but also improves structural lung integrity. This development represents a significant contribution to the achievement of Sustainable Development Goal 3 (SDG 3): Good Health and Well-being, by providing an innovative treatment for a chronic non-communicable respiratory disease, thereby promoting healthier lives for affected individuals.

Clinical Efficacy and Structural Improvements

ASPEN Phase 3 Trial Outcomes

The approval of brensocatib was supported by the ASPEN phase 3 trial, which demonstrated substantial clinical benefits. The trial’s primary outcomes align with key public health objectives central to SDG 3.

• Significant reduction in the rate of pulmonary exacerbations.
• Extended time to the first exacerbation for patients.
• Increased probability of patients remaining exacerbation-free over a one-year period compared to placebo.

CT Substudy Findings on Disease Progression

A substudy of 100 patients from the ASPEN trial provided evidence that brensocatib may halt or reverse disease progression, a critical factor in long-term patient well-being. The findings were analyzed using the BEST-CT and LungQ-BA scoring systems at baseline and 52 weeks.

1. 25-mg Dose Efficacy: This dosage was associated with statistically significant reductions in mucus plugging and an increase in healthy lung parenchyma, suggesting a disease-modifying effect.
2. 10-mg Dose Efficacy: This dosage resulted in significantly smaller bronchus outer diameter measures, indicating a reduction in bronchial wall thickness.
3. Expert Interpretation: Lead author Dr. James D. Chalmers noted that these results suggest brensocatib’s anti-inflammatory activity has downstream effects on mucus, potentially preventing long-term disease progression and supporting the use of the 25-mg dose as a first-line treatment.

Contribution to Sustainable Development Goals (SDGs)

SDG 3: Good Health and Well-being

Brensocatib’s approval directly supports SDG 3 by offering an effective treatment that reduces the burden of a chronic respiratory illness. By mitigating exacerbations and potentially slowing disease progression, the drug enhances the quality of life and well-being for individuals with NCFB.

SDG 9: Industry, Innovation, and Infrastructure

The development of brensocatib, a first-in-class DPP-1 inhibitor, exemplifies the type of scientific research and innovation called for by SDG 9. The partnership between the manufacturer, Insmed, and the clinical research community demonstrates a successful model for fostering innovation in the pharmaceutical sector to address unmet medical needs.

SDG 8 & 10: Decent Work, Economic Growth, and Reduced Inequalities

Chronic diseases like NCFB can impact an individual’s ability to work, affecting economic productivity. An effective, disease-modifying treatment can improve patient health to a degree that supports continued participation in the workforce, contributing to SDG 8. However, as noted by external expert Dr. Daniel A. Salerno, ensuring equitable access is paramount. Issues of cost and payer coverage must be addressed to prevent the treatment from widening health disparities, a key concern of SDG 10.

Future Outlook and Recommendations

Expert Analysis

Dr. Salerno affirmed that the imaging substudy strengthens the case for brensocatib as a disease-modifying agent, moving beyond simple symptom management. While the structural changes observed over one year were modest, they represent a meaningful signal of an effect on disease biology. He emphasized that the clearest benefits are expected in patients with neutrophilic inflammation and a history of exacerbations.

Path Forward for Maximizing Global Health Impact

To fully realize brensocatib’s potential contribution to the SDGs, several steps are necessary. These actions will confirm its long-term benefits and ensure its role in promoting global health equity.

1. Conduct larger, prospectively planned imaging studies with prespecified endpoints to definitively quantify the drug’s structural benefits.
2. Initiate long-term follow-up studies to assess sustained efficacy and safety over extended periods.
3. Gather real-world data on effectiveness, cost-effectiveness, and patient adherence post-launch.
4. Develop strategies in partnership with global health organizations and payers to ensure fair and equitable access for all eligible patient populations, thereby upholding the principles of SDG 10.

Sustainable Development Goals (SDGs) Addressed in the Article

SDG 3: Good Health and Well-being

• The article’s primary focus is on a new medical treatment, brensocatib, for noncystic fibrosis bronchiectasis (NCFB), a chronic non-communicable lung disease. It discusses the drug’s effectiveness in improving patient health outcomes, such as reducing pulmonary exacerbations and improving lung structure, which directly aligns with the goal of ensuring healthy lives and promoting well-being.

SDG 9: Industry, Innovation, and Infrastructure

• The article highlights the process of medical innovation, from research and development to clinical trials and regulatory approval. It details the “ASPEN phase 3 trial” and a subsequent substudy, funded by the manufacturer Insmed. This represents an investment in scientific research and the development of new health technologies, which is a key aspect of SDG 9.

Specific SDG Targets Identified

SDG 3: Good Health and Well-being

1. Target 3.4: By 2030, reduce by one-third premature mortality from non-communicable diseases through prevention and treatment and promote mental health and well-being.
   • The article discusses a new treatment for NCFB, a non-communicable disease. The drug brensocatib is shown to be “disease-modifying,” as it reduces “pulmonary exacerbations,” slows “lung function decline,” and leads to “structural change on imaging.” This directly contributes to the treatment and management of an NCD, aiming to improve long-term health and prevent disease progression.
2. Target 3.8: Achieve universal health coverage, including financial risk protection, access to quality essential health-care services and access to safe, effective, quality and affordable essential medicines and vaccines for all.
   • The FDA approval of brensocatib signifies the availability of a new, effective, and quality medicine. The article also explicitly points to the challenges of achieving universal access by noting that “issues of cost, access, and payer coverage will influence real-world uptake,” directly referencing the affordability and accessibility components of this target.
3. Target 3.b: Support the research and development of vaccines and medicines for the communicable and non-communicable diseases…
   • The entire article is a testament to this target. It describes the research process, from the “ASPEN phase 3 trial” to a specialized “CT substudy,” all focused on developing a new medicine for an NCD. The article mentions that “The study was supported by brensocatib manufacturer Insmed,” which exemplifies private sector support for R&D.

SDG 9: Industry, Innovation, and Infrastructure

1. Target 9.5: Enhance scientific research, upgrade the technological capabilities of industrial sectors in all countries… encouraging innovation and substantially increasing the number of research and development workers… and public and private research and development spending.
   • The development of brensocatib, an innovative “inhibitor of DPP-1,” and the advanced imaging analysis techniques used in the substudy (BEST-CT and LungQ-BA) are clear examples of enhanced scientific research and innovation. The funding of the trial by the manufacturer, Insmed, is a direct instance of private R&D spending aimed at creating new health technologies.

Indicators for Measuring Progress

SDG 3: Good Health and Well-being

• Indicators for Target 3.4:
  • Rate of pulmonary exacerbations: The article explicitly states that brensocatib “significantly reduced pulmonary exacerbations” and “extended the time to the first exacerbation.” This is a direct clinical indicator of improved disease management.
  • Measures of lung structure and function: The article implies that progress can be measured by changes in CT scans. Specific indicators mentioned include “reductions in BEST-CT subscores of bronchiectasis with mucus plugging,” “increased healthy tissue (parenchyma),” and improved “bronchial wall thickness.” These serve as indicators of the drug’s ability to halt or reverse disease progression.
• Indicators for Target 3.8:
  • Availability of new medicines: The “FDA approval” of brensocatib is a clear indicator that a new, quality-assured medicine is available to patients.
  • Medicine accessibility and affordability: The article implies that “cost, access, and payer coverage” are key indicators that will determine the drug’s “real-world uptake” and thus measure progress towards equitable access.

SDG 9: Industry, Innovation, and Infrastructure

• Indicators for Target 9.5:
  • Private sector R&D expenditure: The article provides a specific example of this indicator by stating, “The study was supported by brensocatib manufacturer Insmed.”
  • Output of scientific research: The completion and publication of the “ASPEN phase 3 trial” and the CT substudy, presented at the “American College of Chest Physicians (CHEST) 2025 Annual Meeting,” are indicators of productive research and innovation activities.

Summary of SDGs, Targets, and Indicators

Source: medscape.com