Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for brensocatib for patients with non-cystic fibrosis bronchiectasis. In its Day 60 communication to Insmed, the FDA granted Priority Review to the NDA and set a target action date of August 12, 2025, under the Prescription Drug User Fee Act (PDUFA). At present, the FDA has not indicated whether an advisory committee will be convened to discuss the application. Brensocatib has the potential to become the first and only approved treatment for bronchiectasis and the first in a new class of medicines called dipeptidyl peptidase 1 (DPP1) inhibitors for the treatment of neutrophil-mediated diseases.
“Bronchiectasis is a chronic, progressive disease with no approved treatments, leaving hundreds of thousands of people in the U.S. without an effective way to reduce the pulmonary exacerbations that can lead to serious consequences,” said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. “Brensocatib has the potential to transform the treatment landscape for bronchiectasis and we were pleased to receive the FDA acceptance of our NDA with Priority Review even earlier than anticipated. We look forward to working with the FDA throughout the review process and, pending approval, bringing the first ever bronchiectasis treatment to patients as quickly as possible.”
This NDA is based on data from the landmark Phase 3 ASPEN study, which met its primary endpoint with both dosage strengths of brensocatib achieving statistical and clinical significance for the reduction in the annualized rate of pulmonary exacerbations versus placebo over the 52-week treatment period. Both dosage strengths also met several prespecified exacerbation-related secondary endpoints, including significantly prolonging the time to first exacerbation and significantly increasing the odds of remaining exacerbation-free over the treatment period. Patients treated with brensocatib 25 mg also showed significantly lower lung function decline at week 52 as measured by post-bronchodilator forced expiratory volume over one second.
Brensocatib was well-tolerated in the study. Treatment-emergent adverse events occurring in at least 5.0% of patients treated with either dose of brensocatib and more frequently than in placebo were COVID-19 (15.8%, 20.9%, 15.8%), nasopharyngitis (7.7%, 6.3%, 7.6%), cough (7.0%, 6.1%, 6.4%), and headache (6.7%, 8.5%, and 6.9%) for brensocatib 10 mg, brensocatib 25 mg, and placebo, respectively.
The FDA grants Priority Review to applications for medicines that, if approved, would provide a significant improvement in safety or effectiveness in the treatment of a serious condition. The FDA had previously granted brensocatib Breakthrough Therapy Designation, which is designed to expedite the development and review of therapies that are intended to treat a serious or life-threatening condition, and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on a clinically significant endpoint.
Insmed plans to file regulatory submissions for brensocatib in the EU, UK, and Japan in 2025, with commercial launches anticipated in 2026, pending approval in each territory.
