Amikacin Liposomal Inhalation Suspension: Advances in Application Against Refractory Mycobacterium avium Complex.

Mycobacterium avium complex (MAC) is the most prevalent pathogen among slow-growing Nontuberculous mycobacteria (NTM), and the global incidence of MAC pulmonary disease (MAC-PD) continues to rise steadily. Its intrinsic resistance to conventional anti-mycobacterial agents poses a major therapeutic obstacle. Currently, macrolide-based multidrug regimens remain the standard of care; however, treatment options for macrolide-resistant MAC-PD are critically limited, leading to poor outcomes. Refractory MAC-PD is conventionally defined as persistent sputum culture positivity after at least six months of guideline-based macrolide-containing therapy. As a novel targeted preparation, amikacin liposome inhalation suspension (ALIS) utilizes liposomal encapsulation technology to deliver high and sustained intracellular drug concentrations within alveolar macrophages, while significantly reducing systemic toxic and side effects. It is currently the first and only approved inhaled formulation specifically indicated for refractory MAC-PD, and the addition of ALIS to guideline-based therapy significantly improved sputum culture conversion (Phase III CONVERT trial: 29.0% versus 8.9%,). This review systematically dissects the pharmacological rationale, summarizes pivotal clinical trials, including Phase 2/3 clinical trial data and post-marketing real-world evidence, and further discusses the existing limitations and unresolved clinical challenges. Based on the latest research advances, this review aims to provide a comprehensive and practical reference for clinical decision-making in refractory MAC-PD management.