The US FDA has granted Priority Review to a supplemental New Drug Application (sNDA) from Bridgewater, New Jersey-based Insmed Incorporated (Nasdaq: INSM) for Arikayce (amikacin liposome inhalation suspension), an inhaled liposomal aminoglycoside antibiotic, seeking full approval for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial regimen. The PDUFA target action date is January 28, 2027.
The filing seeks to convert Arikayce's existing FDA accelerated approval — granted in 2018 and restricted to refractory MAC lung disease patients who had failed at least six months of multidrug therapy — into a full approval that would also cover newly diagnosed patients. Insmed said the company also plans to review the data with Japan's Pharmaceuticals and Medical Devices Agency (PMDA) in the second half of 2026 to support potential label expansion there.
The sNDA is supported by results from the Phase IIIb ENCORE study, which enrolled 425 antibiotic-naive adults with a new occurrence of MAC lung infection across 177 global sites. Patients were randomized 1:1 to once-daily Arikayce plus multidrug therapy (azithromycin 250 mg and ethambutol 15 mg/kg) or placebo plus the same regimen for 12 months, followed by three months off treatment. The study met its primary endpoint — change from baseline in Respiratory Symptom Score at Month 13 — and all multiplicity-controlled secondary culture conversion endpoints, with Arikayce-treated patients achieving earlier, greater, and more durable culture conversion than the comparator arm. No new safety signals were observed. ENCORE was also the post-marketing confirmatory trial required by the FDA as a condition of the 2018 accelerated approval.