FDA Approves Brinsupri (Brensocatib), First Targeted Therapy for Non-Cystic Fibrosis Bronchiectasis

The FDA has approved Brinsupri (brensocatib) as the first and only treatment for non-cystic fibrosis bronchiectasis (NCFB), a chronic lung disease affecting about 500,000 people in the US. The once-daily oral therapy comes in 10 mg and 25 mg doses for adults and children aged 12 and older.

It was developed by Insmed, a biopharma company focused on serious and underserved diseases.

What Is Non-Cystic Fibrosis Bronchiectasis?

NCFB is a progressive condition where repeated infections and inflammation permanently widen the airways. This makes it harder to clear mucus and bacteria, leading to ongoing inflammation often driven by neutrophils.

Patients frequently experience flare-ups, or exacerbations, that can result in coughing, mucus buildup, shortness of breath, fatigue and a gradual decline in lung function.

NCFB was once thought to be rare in developed countries, which meant it often went undiagnosed. Advances in imaging, especially high-resolution CT scans, have revealed that it is more common than previously believed.

Developing medicines for NCFB has been challenging as the disease varies widely among patients.

Many patients are diagnosed at advanced stages, which limits the benefits of treatment. Until now, care for NCFB has mostly relied on off-label use of drugs made for other respiratory diseases — particularly antibiotics, mucus-thinning medicines and anti-inflammatory drugs — and the high cost of newer treatments has made them harder to access.

A lack of awareness among healthcare providers and the public has also contributed to underdiagnosis.

How Brensocatib Works — and What the Trials Show

Brensocatib is a first-in-class dipeptidyl peptidase 1 (DPP1) inhibitor that blocks an enzyme needed to activate inflammatory proteins in neutrophils. By reducing the activity of these proteins, it aims to lower airway inflammation and decrease the frequency of exacerbations.

Its approval was based on the Phase III ASPEN and Phase II WILLOW randomized, placebo-controlled studies.

ASPEN included over 1,700 patients worldwide who received either 10 mg or 25 mg of brensocatib, or a placebo once daily for 52 weeks. Both doses met the primary goal, reducing the average number of exacerbations per year by 21.1% for the 10 mg dose and 19.4% for the 25 mg dose compared to placebo.

Secondary goals were also achieved, such as longer time to first exacerbation and a higher percentage of patients who remained exacerbation-free during treatment.

Those on the 25 mg dose showed less decline in post-bronchodilator forced expiratory volume in one second (FEV₁) — a standard measure of lung capacity tested after using an inhaler — at Week 52.

The 24-week WILLOW study supported these findings. In both trials, brensocatib was generally well-tolerated.

The most common side effects in at least 2% of participants in ASPEN included upper respiratory tract infection, headache, rash, dry skin, hyperkeratosis and high blood pressure.

Less common issues included mild liver enzyme elevations, low rates of skin cancer and hair loss.

When Will Brensocatib be Available?

Brinsupri is now available in the US through a specialty pharmacy network that includes PANTHERx Rare Pharmacy and Amber Specialty Pharmacy, both of which provide patient access and support services.

Regulatory reviews are underway in the European Union (EU) and the UK, with a filing planned in Japan for 2025. Additional launches are expected in 2026, pending approvals in those regions.

The approval of Brinsupri comes as other companies work on treatments for NCFB. Days earlier, Expedition Therapeutics announced a deal worth up to $645 million with Shanghai Fosun Pharmaceutical Group for rights to XH-S004, an oral DPP1 inhibitor in Phase II testing for NCFB and Phase Ib for COPD, outside mainland China, Hong Kong and Macau.

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