EU committee backs the first medicine for non-CF bronchiectasis
Brensocatib cleared CHMP on an accelerated timetable with a 19.4% cut in exacerbation rate. The same October meeting issued a negative opinion on belumosudil and kept a sickle cell drug suspended.
The European Medicines Agency's human medicines committee recommended two new medicines for approval at its 13–16 October meeting, and one of them would be the first authorised treatment in the EU for a lung disease that currently has none [s2].
Brinsupri (brensocatib), 25 mg tablets, received a positive opinion for non-cystic fibrosis bronchiectasis in patients aged 12 and over who have had two or more exacerbations in the prior 12 months [s1]. EMA estimates between 400,000 and three million people in the EU have the condition, and states plainly that there are currently no authorised medicines for it — patients rely on airway clearance and receive antibiotics and anti-inflammatory medicines [s1].
What the disease costs patients now
Non-cystic fibrosis bronchiectasis is a chronic, progressive disease in which the airways are damaged, producing chronic cough and airflow obstruction from abnormal mucus production [s1]. It is driven by repeated infection and inflammation and can be triggered by respiratory infections, autoimmune diseases, and immunodeficiency disorders [s1].
Patients typically experience between one and four exacerbations a year, and those exacerbations are associated with progressive lung function decline, reduced quality of life, and increased mortality [s1]. Slowing the exacerbation rate is therefore the endpoint the whole development programme was built around.
The mechanism, and what it delivered
Brensocatib inhibits dipeptidyl peptidase 1, an enzyme involved in activating neutrophils [s1]. In bronchiectasis, repeated neutrophil activation causes excessive release of neutrophil serine proteases, which damage the airway wall, drive mucus production and sustain inflammation [s1]. By inhibiting DPP1, brensocatib prevents those proteases from being activated [s1].
EMA's recommendation rests on a randomised, double-blind, placebo-controlled trial in 1,767 patients [s1]. Patients on the 25 mg dose had a 19.4 percent reduction in the annual rate of pulmonary exacerbations and a 14-week delay in median time to first exacerbation, with a significantly higher proportion remaining exacerbation-free at week 52 [s1].
The published phase 3 results, which appeared in April, give the underlying figures [s3]. In that report 1,721 patients — 1,680 adults and 41 adolescents — underwent randomisation and received brensocatib 10 mg, brensocatib 25 mg, or placebo [s3]. The annualised exacerbation rate was 1.02 in the 10 mg group, 1.04 in the 25 mg group, and 1.29 on placebo, giving rate ratios of 0.79 (95% CI 0.68–0.92; adjusted P = 0.004) and 0.81 (0.69–0.94; adjusted P = 0.005) respectively [s3]. Hazard ratios for time to first exacerbation were 0.81 (0.70–0.95) and 0.83 (0.70–0.97) [s3]. In each brensocatib group 48.5 percent of patients remained exacerbation-free at week 52, against 40.3 percent on placebo [s3].
That is a real effect on a hard endpoint in a disease with no approved therapy. It is also modest in absolute terms, and the published annualised rates are the clearest way to see it: 1.02 and 1.04 on treatment against 1.29 on placebo [s3]. Most treated patients still had an exacerbation within the year.
The side effects worth knowing about
The most common side effects EMA lists are headache, gingival and periodontal disease, and skin problems including hyperkeratosis, dermatitis, rashes and dry skin [s1]. The gum and skin effects are mechanistically expected — neutrophil serine proteases are not confined to the lung — and they are the trade-off in a medicine intended for long-term use.
How it got here quickly
Brinsupri was supported through EMA's PRIME scheme, having been granted eligibility on 27 February 2020 for exactly this indication and patient definition [s1]. CHMP reviewed the marketing authorisation application under an accelerated timetable on the grounds that the medicine is of major public health interest [s1]. The applicant is Insmed Netherlands B.V. [s1].
The rest of the October docket
The second positive opinion went to Wayrilz (rilzabrutinib), from Sanofi B.V., for immune thrombocytopenia in adults refractory to other treatments, with orphan designation [s2].
The committee recommended against granting a marketing authorisation for Rezurock (belumosudil), intended for chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy [s2]. It also confirmed, on re-examination, its earlier recommendation not to consider deutetrabenazine — the active substance in Austedo, for tardive dyskinesia — as a new active substance [s2].
Eight already-authorised medicines were recommended for extensions of indication: Breyanzi, Cejemly, Gazyvaro, Libtayo, Paxlovid, Pyrukynd, Tremfya and Scemblix [s2]. One initial application was withdrawn: Hydrocortisone Aguettant, developed to prevent bronchopulmonary dysplasia in preterm infants born before 28 weeks of gestation [s2].
The committee also recommended that the marketing authorisation for the sickle cell disease medicine Oxbryta remain suspended, following interim measures taken in September 2024 [s2].
The 2025 running totals
October's meeting brought the year's cumulative CHMP figures to 87 positive opinions on new medicines, six negative opinions, 73 positive opinions on extensions of indication, and 18 withdrawn applications [s2]. October itself contributed two positives — one non-orphan, one orphan — one negative, eight extensions and one withdrawal [s2].
What happens next
A CHMP opinion is not an authorisation. The opinion goes to the European Commission, which issues the legally binding decision applicable across member states [s1]. After that, each member state decides separately on price and reimbursement, taking into account the medicine's role within its own health system [s1] — which is where the practical question of who actually gets brensocatib, and when, will be settled.
Sources
- First treatment for serious chronic lung disease — European Medicines Agency, 17 October 2025
- Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 13-16 October 2025 — European Medicines Agency, 17 October 2025
- Phase 3 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis — The New England Journal of Medicine, 1 April 2025
Sources
- First treatment for serious chronic lung disease — European Medicines Agency , October 17, 2025
- Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 13-16 October 2025 — European Medicines Agency , October 17, 2025
- Phase 3 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis — The New England Journal of Medicine , April 1, 2025
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