FDA Sets Review Date for Chronic Lung Disease Therapy Brensocatib
First-in-class brensocatib has the potential to be the first approved treatment for bronchiectasis.
The FDA has accepted Insmed Incorporated’s new drug application (NDA) for brensocatib to treat patients with non–cystic fibrosis bronchiectasis. Regulators have granted priority review and set a target action date of August 12, 2025, under the Prescription Drug User Fee Act.1
Bronchiectasis is a serious chronic lung disease where the airways (bronchi) become permanently dilated due to a cycle of infection, inflammation, and resulting lung tissue damage. This leads to frequent pulmonary exacerbations that often require treatment with an antibiotic and/or hospitalizations. According to a recent Insmed news release, approximately 500,000 patients in the United States have been diagnosed with bronchiectasis.1
“Bronchiectasis is a chronic, progressive disease with no approved treatments, leaving hundreds of thousands of people in the US without an effective way to reduce the pulmonary exacerbations that can lead to serious consequences,” Martina Flammer, MD, MBA, chief medical officer of Insmed, said in the news release.1
Brensocatib could become the first approved treatment for bronchiectasis. | image credit: TripleP Studio / stock.adobe.com
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Brensocatib could become the first approved treatment for bronchiectasis and the first in a new class of medications called dipeptidyl-peptidase-1 (DPP-1) inhibitors for the treatment of neutrophil-mediated diseases. According to the news release, “DPP-1 is an enzyme responsible for activating neutrophil serine proteases (NSPs)...in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation.”1
Insmed’s NDA is based on data from the phase 3 ASPEN study (NCT04594369). The trial met its primary end point, “with both dosage strengths of brensocatib achieving statistical and clinical significance for the reduction in the annualized rate of pulmonary exacerbations vs placebo over the 52-week treatment period,” according to the news release. The primary efficacy analysis in ASPEN included data from 1680 adult patients and 41 adolescent patients with non–cystic fibrosis bronchiectasis.1
In a 2024 news release, Insmed noted that the annualized rate of pulmonary exacerbations also favored brensocatib at both the 10- and 25-mg doses vs placebo for most subgroups. In a separate analysis, “least squares mean difference for brensocatib 25 mg demonstrated a reduced decline in post-bronchodilator [forced expiratory volume in the first second of expiration (FEV1)] at week 52 vs placebo for all prespecified subgroups. Results for patients treated with brensocatib 10 mg among subgroups were aligned with the overall trial population results.” FEV1 is a common measure of airway obstruction.2
Treatment-emergent adverse events occurring in at least 5% of patients treated with either dose of brensocatib and more frequently than with placebo were COVID-19, nasopharyngitis, cough, and headache.2
Insmed is also conducting the phase 2b BiRCh trial of brensocatib in patients with chronic rhinosinusitis without nasal polyps and noted in a news release that it anticipates providing top-line data from the study in the second half of 2025.3 In addition, the company is conducting the phase 2b CEDAR study of brensocatib in patients with hidradenitis suppurativa, a skin condition that causes abscesses and scarring.4
This article originally appeared on Formulary Watch. To read these stories and more, download the PDF of the Total Pharmacy April issue here.
References
1. FDA grants priority review to Insmed’s brensocatib for treatment of bronchiectasis with PDUFA Target Action Date set for August 12, 2025. News release. Insmed Incorporated. February 6, 2025. Accessed February 21, 2025. https://investor.insmed.com/2025-02-06-FDAGrants-Priority-Review-to-Insmeds-Brensocatib-for-Treatment-of-Bronchiectasis-with-PDUFA-Target-Action-Date-Set-for-August-12,--2025
2. New subgroup analyses from landmark phase 3 ASPEN study of brensocatib in patients with bronchiectasis demonstrate consistency with positive overall trial population results. News release. Insmed Incorporated. October 8, 2024. Accessed February 21, 2025. https://investor.insmed.com/2024-10-08-New-Subgroup-Analyses-from-Landmark-Phase-3-ASPEN-Study-of-Brensocatib-in-Patients-with-Bronchiectasis-Demonstrate-Consistency-with-Positive-Overall-Trial-Population-Results
3. Insmed reports first-quarter 2024 financial results and provides business update. News release. Insmed Incorporated. May 9, 2024. Accessed February 21, 2025. https://investor.insmed.com/2024-05-09-Insmed-Reports-First-Quarter-2024-Financial-Results-and-Provides-Business-Update
4. Insmed reports fourth-quarter and full-year 2024 financial results and provides business update. News release. Insmed Incorporated. February 20, 2025. Accessed February 21, 2025. https://investor.insmed.com/2025-02-20-Insmed-Reports-Fourth-Quarter-and-Full-Year-2024-Financial-Results-and-Provides-Business-Update
