FDA approves a new antibacterial drug to treat a serious lung disease

FDA approved on September 28 amikacin liposome inhalation suspension (Arikayce—Insmed) for the treatment of lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC), in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease).

FDA approved on September 28 amikacin liposome inhalation suspension (Arikayce—Insmed) for the treatment of lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC), in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease). The condition—a type of nontuberculous mycobacteria often found in water and soil—causes symptoms including persistent cough, fatigue, weight loss, night sweats, and sometimes shortness of breath and coughing up of blood. FDA Commissioner Scott Gottlieb, MD, noted: "This approval is the first time a drug is being approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, and it marks an important policy milestone. This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies." The drug was also approved under the Accelerated Approval pathway. FDA's approval of amikacin liposome inhalation suspension was based on achieving three consecutive negative monthly sputum cultures by month 6 of treatment. The drug's sponsor will be required to conduct an additional, post-market study to describe amikacin liposome inhalation suspension's clinical benefits. The drug's prescribing information includes a boxed warning about the higher risk of respiratory conditions including hypersensitivity pneumonitis, bronchospasm, exacerbation of underlying lung disease, and hemoptysis that have led to hospitalizations in some cases. Other common adverse effects in patients taking the drug were dysphonia, cough, ototoxicity, upper airway irritation, musculoskeletal pain, fatigue, diarrhea, and nausea.