01—Abstract
Summary
Summary of the 8-K filed February 6, 2025. Cited figures are marked; the original filing is the authoritative source.
Insmed Incorporated (Nasdaq: INSM) announced on February 6, 2025 that the U.S. Food and Drug Administration has accepted for review its New Drug Application for brensocatib for patients with non-cystic fibrosis bronchiectasis.
The FDA granted Priority Review and set a Prescription Drug User Fee Act target action date of August 12, 2025. The company said the FDA has not indicated whether an advisory committee will be convened. The application is based on the Phase 3 ASPEN study, in which both dosage strengths of brensocatib (10 mg and 25 mg) met the primary endpoint of reducing the annualized rate of pulmonary exacerbations versus placebo over 52 weeks.
Both doses also met several prespecified exacerbation-related secondary endpoints, and the 25 mg dose showed significantly lower lung function decline at week 52 as measured by post-bronchodilator FEV1. Treatment-emergent adverse events occurring in at least 5.0%a of patients more frequently than placebo included COVID-19, nasopharyngitis, cough, and headache. The FDA had previously granted brensocatib Breakthrough Therapy Designation.
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. The filing describes brensocatib as an investigational drug that has not been approved for any indication in any jurisdiction.
02—Key points
Key Highlights
- FDA accepted the NDA for brensocatib for non-cystic fibrosis bronchiectasis and granted Priority Review.
- PDUFA target action date set for August 12, 2025.
- The company said FDA has not indicated whether an advisory committee will be convened.
- Phase 3 ASPEN study met its primary endpoint for both 10 mg and 25 mg doses in reducing the annualized rate of pulmonary exacerbations versus placebo over 52 weeks.
- Brensocatib would be the first and only approved treatment for bronchiectasis and the first DPP1 inhibitor, if approved.
- Brensocatib previously received Breakthrough Therapy Designation from the FDA.
- EU, UK, and Japan regulatory submissions planned for 2025, with commercial launches anticipated in 2026, pending approval.
03—Questions
Frequently Asked Questions
What regulatory milestone did Insmed announce?
The FDA accepted the company's New Drug Application for brensocatib for patients with non-cystic fibrosis bronchiectasis, granted Priority Review, and set a PDUFA target action date of August 12, 2025.
What data support the application?
The application is based on the Phase 3 ASPEN study. Both dosage strengths met the primary endpoint of reducing the annualized rate of pulmonary exacerbations versus placebo over 52 weeks, and both met several prespecified exacerbation-related secondary endpoints. The 25 mg dose also showed significantly lower lung function decline at week 52 as measured by post-bronchodilator FEV1.
What safety information did the company disclose?
The company reported that brensocatib was well-tolerated. Treatment-emergent adverse events occurring in at least 5.0% of patients and more frequently than placebo included COVID-19 (15.8% for 10 mg, 20.9% for 25 mg, 15.8% for placebo), nasopharyngitis, cough, and headache.
Does brensocatib have an approved status in any market?
No. The filing states that brensocatib is an investigational drug that has not been approved for any indication in any jurisdiction.
What are the next steps outside the United States?
Insmed plans to file regulatory submissions in the EU, UK, and Japan in 2025, with commercial launches anticipated in 2026, pending approval in each territory.
04—Continue