Pfor those who live with them bronchiectasis unrelated to cystic fibrosis, the 2025 will remain a turning point year. After decades in which this chronic respiratory disease remained on the margins of public interest, the European Commission has approved BRINSUPRI® (brensocatib)the first therapy specifically designed to reduce exacerbationsthat is, the exacerbationwhich mark the course of the pathology and accelerate its worsening. At the center of this change there are two crucial voices: Steven HuysseHead of EMEA Medical Affairs at Insmedand the Professor Stefano Alibertione of Europe’s leading experts on bronchiectasis. We had the chance to interview them for the readers of iODonna, here’s what they told us.
Bronchiectasis, 2025 is the turning point year
A goal that doesn’t just concern patients already diagnosed, estimated at around 600,000 in the European Union, but also that vast number of people, up to two million, who may suffer without knowing it, confused by alternative diagnoses or by symptoms that drag on over time. The European green light, which arrived after an accelerated assessment (an evaluation procedure that reduces the time for the approval of new drugs) of theEMArecognizes not only theclinical urgency but also the solidity of the data coming from the ASPEN and WILLOW studies, published in New England Journal of Medicine. For the first time, a pathology often considered “orphan” receives targeted treatment.
With Brindsupri can we talk about a historical turning point?
Full Professor of Respiratory Diseases at the Department of Biomedical Sciences of Humanitas University. Professor Aliberti is director of the European (EMBARC) and Italian (IRIDE) bronchiectasis register and author of the latest European guidelines on the management of the disease. «Yes, this is truly a historic turning point. For the first time there is a therapy approved specifically for bronchiectasis not due to cystic fibrosis. So far we have used off-label therapies. The FDA and EMA approval represents a decisive moment, awaited for decades both by patients and the scientific community. This for the first time pathology receives full dignity and regulatory attention. Bronchiectasis is no longer considered an “orphan” condition, but one chronic respiratory disease that deserves dedicated therapies».
What does “reduction of exacerbations” mean?
«Each flare-up is like an attack on the lung: increased cough, more phlegm, more infections. Each episode can leave a mark on the airways and accelerate the worsening of respiratory function. A therapy capable of reducing the number of these crises is very important news. Reducing exacerbations means reducing symptoms, improving disease control, preserve lung function and resort to fewer antibiotics, fewer hospitalizations and more wellness days”, clarifies Professor Aliberti.
Why does the diagnosis arrive so late in Italy?
Coe explains Professor Aliberti, «they can pass 9 or 10 years from the onset of symptoms before a patient arrives at a specialized clinic. In this period they are often formulated diagnosis of asthma or COPD. Territorial medicine has a fundamental role in‘intercept alarm signals: daily cough and phlegm, frequent bronchitis or pneumonia. You need to request a high-resolution chest CT scan. If the suspicion is confirmed, the patient should be sent to the pulmonologist.”
When to ask for a specialist evaluation?
«Chronic cough, daily production of phlegm and recurrent infections are signs that should raise suspicion of the disease. Even a single episode of blood in the phlegm is not to be ignored. High resolution CT scan allows diagnosis. Once the radiological confirmation has been obtained, the pulmonologist’s evaluation is essential to set up adequate management and prevent progressive deterioration”, recommends Professor Aliberti.
Now that Brinsupri has been approved, what are the priorities to ensure access and support for patients with bronchiectasis?
Steven HuysseHead of EMEA Medical Affairs at Insmedunderlines that the initial efforts will focus on pulmonologists and lung specialists, central figures in the diagnosis, and that communication will also extend to the public. «The Bronchiectasis is the third most common chronic airway disease after asthma and COPDbut one of the most overlooked. With the approval of BRINSUPRI by the European Commission we will work with the competent authorities to guarantee access to reimbursement for eligible patients. At the same time, we are committed to improving awareness and understanding of the disease within the medical community.”
What elements of the clinical data contributed to the EMA’s accelerated approval?
«The NCFBs represent one chronic and progressive disease characterized by recurrent exacerbations and deterioration of lung function. Previously there were no approved therapies, only symptomatic treatments. Brensocatib represents a therapeutic innovation because it addresses inflammation, one of the fundamental causes of the disease.” The accelerated approval, explains Huysse, reflects both the broad therapeutic need and the robustness of the evidence.
What does this approval mean for Insmed’s strategy and for patients?
«Our mission is to bring new therapies to patient communities who do not have access to necessary care. We focus on serious diseases that are typically overlooked. Our pipeline includes several potential respiratory indications and our research approach aims to serve communities with the greatest needs,” explains Dr Huysse.
After years in which patients felt invisible, what does this milestone represent for you?
«It is deeply meaningful to know that our efforts are helping to improve patients’ lives. We are grateful to the patients, clinicians and partners who made this achievement possible. Now the challenge is to ensure that the treatment reaches those who need it, because innovation is only valuable if it reaches the patient”, he adds Huysse.
A future that finally takes shape
The arrival of BRINSUPRI opens a new season for a community of patients who have felt on the margins for too long. Science makes its stride, but now the commitment passes to the institutions, to local medicine and to patient awareness. As Huysse reminds us, “innovation is only truly valuable when it reaches the patient”.
And today, for the first time, bronchiectasis has a dedicated therapy. A new therapeutic path, which brings hope.