Insmed Incorporated announced that the U.S. Food and Drug Administration (FDA) has approved BRINSUPRI™ (brensocatib), a new oral, once-daily medication for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in adults and adolescents aged 12 and older. This is a significant milestone, as BRINSUPRI is the first and only approved treatment for NCFB, a progressive and chronic lung disease affecting approximately 500,000 patients in the U.S.

Unlike other respiratory illnesses that cause airways to narrow, NCFB leads to the permanent widening of the airways, making it difficult to clear mucus and bacteria. This results in persistent infection and inflammation, and frequent symptom flare-ups, known as exacerbations, which can lead to permanent lung damage and a decline in lung function over time. NCFB has a profound impact on patients' physical and emotional well-being, and until now, there were no approved therapies specifically for the condition.

BRINSUPRI is a first-in-class dipeptidyl peptidase 1 (DPP1) inhibitor. It works by targeting and inhibiting the activation of key enzymes within neutrophils, a type of white blood cell. This action helps to reduce the chronic inflammation that is a primary driver of NCFB. By addressing this underlying inflammatory process, the drug is designed to reduce the frequency and severity of exacerbations.

The FDA approval is based on data from the Phase 3 ASPEN and Phase 2 WILLOW studies. The ASPEN trial was a global, placebo-controlled study that demonstrated the efficacy and safety of BRINSUPRI. Patients taking the 10 mg and 25 mg doses of BRINSUPRI experienced a 21.1% and 19.4% reduction, respectively, in the annual rate of exacerbations compared to the placebo group. Both doses of BRINSUPRI significantly prolonged the time to the first exacerbation and increased the proportion of patients who remained exacerbation-free throughout the treatment period. The 25 mg dose of BRINSUPRI also showed a statistically significant reduction in the decline of lung function over 52 weeks, as measured by forced expiratory volume in one second (FEV₁). The most common side effects observed in the ASPEN trial included upper respiratory tract infection, headache, rash, dry skin, and hypertension. The WILLOW study, which evaluated the drug over a shorter period, showed a similar safety profile, though it also noted a higher incidence of gingival and periodontal adverse reactions.

BRINSUPRI is now available by prescription in the U.S. through a network of specialty pharmacies. Insmed has also launched the inLighten Patient Support Program to help eligible patients with financial assistance and educational resources. Insmed has also submitted applications for brensocatib to regulatory agencies in Europe and the UK, with plans to file in Japan in 2025. This suggests that the company anticipates a global launch of the drug in 2026, pending approvals in those regions. This new treatment offers hope for the millions of people worldwide living with this debilitating disease.