Bridgewater, N.J. – September 21, 2026 -- The U.S. Food and Drug Administration has accepted Insmed Incorporated's supplemental New Drug Application (sNDA) for full approval of ARIKAYCE (amikacin liposome inhalation suspension) to treat Mycobacterium avium complex (MAC) lung disease, granting Priority Review with a PDUFA target action date of January 28, 2027.
FDA sets January 28, 2027 decision date under Priority Review
The Priority Review designation applies to therapies that could offer significant improvement in safety or efficacy for serious conditions. Insmed said full approval would allow ARIKAYCE, administered via PARI Pharma's Lamira Nebulizer System, to reach patients earlier in their disease course, including newly diagnosed cases and those with recurrent infections.
Phase 3b ENCORE trial of 425 patients meets primary endpoint
The sNDA is supported by the Phase 3b ENCORE study, which enrolled 425 patients across 177 global sites, with 82.4% experiencing a first MAC infection and 17.6% a second or third occurrence. Patients received once-daily ARIKAYCE plus multidrug therapy (n=213) or placebo plus multidrug therapy (n=212) for 12 months, followed by a three-month treatment-free period. The study met its primary endpoint—change in Respiratory Symptom Score at Month 13—and multiplicity-controlled secondary culture conversion endpoints, with ARIKAYCE-treated patients achieving earlier, greater and more durable culture conversion; no new safety signals emerged.
Approval would satisfy 2018 accelerated approval post-marketing requirement
ARIKAYCE received FDA accelerated approval in 2018 as the first product cleared under the Limited Population Pathway for Antibacterial and Antifungal Drugs. ENCORE was the pivotal study designed to fulfill the post-marketing requirement tied to that accelerated approval. "The FDA's acceptance of this application marks a pivotal moment in our ambition to change the treatment paradigm for MAC lung disease," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed.
Insmed to pursue Japan label expansion via PMDA review in Q4 2026
Insmed plans to submit the ENCORE data to Japan's Pharmaceuticals and Medical Devices Agency in the fourth quarter of 2026 to support a potential label expansion in that market, where ARIKAYCE is already approved as ARIKAYCE inhalation 590 mg.