Insmed ARIKAYCE (amikacin liposomal inhalation suspension) benefits patients with rare bacterial lung disease caused by Mycobacterium avium complex (MAC), study shows[1][2][3]. ARIKAYCE received accelerated approval from the FDA in September 2018 for the treatment of adult patients with refractory MAC lung disease who have not had negative sputum cultures after at least 6 months of multidrug therapy and have limited alternatives[1][3][4]. The phase 3 ARISE study confirmed that the respiratory system domain of the QOL-B questionnaire is an effective tool for assessing the quality of life of patients with MAC lung disease who have not yet started antibiotics[2][3]. Insmed plans to use this tool as the primary endpoint in the ENCORE study and propose it to the FDA without changes[2]. The treatment is inhaled once daily using the Lamira Nebulizer system, which directly targets lung macrophages and limits systemic exposure[2][3][6]. Insmed hopes to turn accelerated approval into full approval based on data from clinical trials[4]. In clinical trials, adverse events such as hypersensitivity pneumonitis (3.1% vs. 0%), hemoptysis (17.9% vs. 12.5%), and bronchospasm (28.7% vs. 10.7%) occurred more frequently with ARIKAYCE plus standard therapy versus standard therapy alone[1][4][7].