Insmed's Brensocatib NDA Accepted by FDA with Priority Review; Anticipated Launch in Q3 2025
Key Insights
The FDA has accepted Insmed's New Drug Application for brensocatib in bronchiectasis (search), granting Priority Review with a PDUFA target action date of August 12, 2025.
Insmed anticipates launching brensocatib in the U.S. in the third quarter of 2025, pending FDA approval, with regulatory submissions planned for the EU, UK, and Japan in 2025.
ARIKAYCE global revenue grew 19% in 2024, and Insmed projects 2025 revenues between $405 million and $425 million, representing double-digit growth.
Insmed Incorporated (Nasdaq: INSM) is poised for a potentially transformative year as the FDA accepts its New Drug Application (NDA) for brensocatib, a novel oral inhibitor of dipeptidyl peptidase 1 (DPP1 (search)), for the treatment of bronchiectasis (search). The FDA granted the application Priority Review, setting a Prescription Drug User Fee Act (PDUFA) target action date of August 12, 2025. This regulatory milestone, announced alongside Insmed's fourth-quarter and full-year 2024 financial results, signals a potential new treatment option for patients with this chronic respiratory condition. Insmed anticipates a U.S. launch in the third quarter of 2025, pending approval. Regulatory submissions in the EU, UK, and Japan are planned for 2025, with commercial launches anticipated in 2026, pending approval in each territory.
Brensocatib: A Novel Approach to Bronchiectasis Treatment
Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1 (search)). DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase. By inhibiting DPP1, brensocatib aims to reduce the damaging effects of excessive active NSPs in the lungs of patients with bronchiectasis (search). Bronchiectasis is a chronic condition in which the bronchioles become permanently damaged and widened, leading to mucus buildup and increased susceptibility to infection.
Insmed is also evaluating brensocatib in other neutrophil-mediated diseases. The Phase 2b BiRCh study of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) (search) remains on track to report topline results by the end of 2025. The company has initiated dosing in the Phase 2b CEDAR study of brensocatib in patients with hidradenitis suppurativa (HS) (search).
ARIKAYCE Revenue Growth and ENCORE Trial
Insmed's existing product, ARIKAYCE (amikacin liposome inhalation suspension), continues to drive revenue growth. Global ARIKAYCE revenue grew 19% in 2024 compared to 2023, reflecting continued strong growth in the U.S., Japan, and Europe. The company anticipates 2025 global ARIKAYCE revenues to be between $405 million and $425 million, representing between 11% and 17% year-over-year growth compared to 2024.
Enrollment is complete in the Phase 3 ENCORE study for patients with newly diagnosed or recurrent Mycobacterium avium complex (MAC) lung disease (search) who had not started antibiotics. Total enrollment in the study was 425 patients. The company continues to anticipate a topline readout for ENCORE in the first quarter of 2026, with the submission of a supplementary new drug application (sNDA) for ARIKAYCE in all patients with MAC lung disease in the U.S. expected later in 2026.
Advancing the Pipeline: TPIP and Gene Therapy
Insmed is also advancing its pipeline with treprostinil palmitil inhalation powder (TPIP) (search) and gene therapy programs. Insmed presented additional lung imaging data from the Phase 2a study of TPIP in pulmonary hypertension associated with interstitial lung disease (PH-ILD) (search) at the Pulmonary Vascular Research Institute's 2025 Annual World Congress. The company plans to initiate a Phase 3 study of TPIP in patients with PH-ILD in the second half of 2025. Enrollment in the Phase 2b study of TPIP in pulmonary arterial hypertension (PAH) (search) completed in December 2024, with 102 patients randomized. Insmed continues to anticipate topline data from the study in the middle of 2025.
The company received clearance from the FDA for its investigational new drug (IND) application for INS1201, an intrathecally-delivered gene therapy for patients with Duchenne muscular dystrophy (DMD) (search), in December 2024. Insmed plans to initiate a clinical study of INS1201 in the first half of 2025.