Results of a large, global clinical trial spanning five continents with over 1,700 patients with bronchiectasis, published this April in the New England Journal of Medicine, demonstrated benefits of an investigational, once-a-day pill called brensocatib as a therapy for the chronic lung condition.
The clinical trial findings are important, as there are currently no FDA-approved medications for bronchiectasis, a chronic condition with persistent lung airway inflammation and infection. Bronchiectasis can often stem from various injuries to the airways causing the ‘bronchial’ tubes leading to the lungs to become permanently enlarged, and more prone to infection and chronic inflammation.
Symptoms of bronchiectasis include chronic cough with sputum (mucous) production, shortness of breath and fatigue. Acute exacerbations of the debilitating condition experienced by patients are characterized by worsening of the cough and sputum production, often with fever, shortness of breath or chest pain and further impair patient quality of life. Severe exacerbations may result in hospitalization and permanent loss of lung function.
Bronchiectasis impacts up to 500,000 Americans, but there is often misdiagnosis or delayed diagnosis as the condition can present similarly to other pulmonary conditions such as COPD or asthma.
Senior study author Dr. Mark Metersky of UConn School of Medicine served on the Steering Committee for the global, multi-center, randomized clinical trial and was principal investigator for UConn School of Medicine’s clinical trial site. Metersky specializes in bronchiectasis care and is the longtime director of UConn’s dedicated Center for Bronchiectasis Care at UConn Health in Farmington, Connecticut.
Most bronchiectasis patients experience loss of lung function over time due to the irreversible damage caused by the progressive disease. But in this large, international, randomized ASPEN trial which included 1,721 patients, the new DPP-1 inhibitor medication targeting inflammation in either a 10 mg or 25 mg pill dose versus placebo over a 52-week period was shown to significantly lower the annualized rate of exacerbations in patients taking either drug dose while also slowing their loss of lung function. Also, the authors report that in each brensocatib group nearly half (48.5%) of patients remained exacerbation-free one year later at week 52.
“Patients with bronchiectasis have impaired quality of life,” shared Metersky who personally cares for well over 100 patients with the condition at UConn Health. “The study results suggests that brensocatib will help many patients living with bronchiectasis. Bronchiectasis patients’ quality of life was measured throughout the study and improved in patients who received the drug.”
“Pulmonary exacerbations of bronchiectasis can last days or weeks and preventing them is important,” stresses Metersky. “However, this drug resulted in improved quality of life even when patients were not suffering from an exacerbation, providing hope for so many patients suffering with daily symptoms.”
Metersky concludes, “This is a very promising new treatment and likely will be the first-ever FDA-approved treatment for bronchiectasis.”
Insmed, Inc. is the drug’s manufacturer and sponsored the clinical trial.
