An Investigational Treatment for Serious Chronic Lung Infections
ARIKAYCE™ (Liposomal Amikacin for Inhalation, or LAI) is an investigational drug comprising the antibiotic amikacin in Insmed’s proprietary liposomal technology formulation. ARIKAYCE is being investigated for treatment of serious lung infections such as those caused by NTM, through delivery of antibiotic directly to the site of the lung infection.
An Investigational Treatment for Serious Chronic Lung Infections
The active ingredient, amikacin, is an antibiotic used to treat a broad range of gram-negative infections. Amikacin is in the aminoglycoside class of antibiotics and is currently delivered intravenously or intramuscularly.

What Are Liposomes?

Liposomes are microscopic membrane shells that contain water. In a liposome drug delivery system, water-soluble drugs, like amikacin, are located in the liposome’s water core. The goal of a liposome delivery system, which is currently investigational, is to deliver medicine directly to the site of the lung infection.

Proprietary Liposomal Technology
Insmed’s proprietary liposomal technology is designed specifically for delivery of pharmaceuticals to the lung.
Proprietary Liposomal Technology
Insmed’s liposomal technology aims to both retain amikacin in the lung, enabling once-daily dosing, and may allow for LAI to gain close access to bacteria within infected alveolar macrophages (immune cells in the lung), as in the case of NTM lung infections. LAI liposomes are readily taken up by macrophages whose normal function is to “swallow” inhaled particles.1,2 In so doing, amikacin is delivered where it is needed most.

The inhalation technology of LAI provides for delivery of amikacin directly to the site of infection in the lungs, minimizing systemic exposure to other organs.

The Role of the Liposome
In NTM lung infections, the infecting bacteria invade and multiply chiefly within macrophages.3 When amikacin (non-liposomal)—which is currently only approved for intravenous or intramuscular use—is delivered via inhalation, it does not achieve the same level of intracellular delivery to the lung’s macrophages as does LAI (animal data on file). Moreover, the materials found in a patient’s mucus and biofilms that are produced by bacteria to protect themselves have negative charges.4-6 Because opposite charges attract each other, positively charged antibiotics like amikacin, bind to the negatively charged compounds.7-9 This prevents effective penetration of positively charged antibiotic drugs into the spaces in which the bacteria are located.7-9

With LAI, amikacin is packaged into liposomes at a high concentration and consequently uptake of these structures by macrophages results in efficient delivery of drug at what are expected to be locally high levels.2 Therefore, the particle nature of the liposomes is key to the effect of ARIKAYCE.


1Malinin V, Neville ME, Eagle G, Perkins W, Gupta R. Lung distribution and clearance of low- or high-dose dual-labeled liposomal amikacin for inhalation in rats dosed once daily for 28 days. Poster presented at: 2014 North American Cystic Fibrosis Conference; October 9-11, 2014; Atlanta, Georgia.

2Rose SJ, Neville ME, Gupta R, Bermudez LE (2014) Delivery of Aerosolized Liposomal Amikacin as a Novel Approach for the Treatment of Nontuberculous Mycobacteria in an Experimental Model of Pulmonary Infection. PLoS ONE 9(9): e108703. doi:10.1371/journal.pone.0108703.

3Crowle AJ, Tsang AY, Vatter AE, May MH. Comparison of 15 laboratory and patient-derived strains of Mycobacterium avium for ability to infect and multiply in cultured human macrophages. J Clin Microbiology. 1986;24(5):812-21.

4Groneberg DA, Eynott PR, Oate T, Lim S, Wu R, Carlstedt I, Nicholson AG, Chung KF. Expression of MUC5AC and MUC5B mucins in normal and cystic fibrosis lung. Respiratory Medicine. 2002;96(2):81-6.

5Bansil R, Turner BS. Mucin structure, aggregation, physiologic functions and biomedical applications. Current Opinion in Colloid and Interface Science. 2006;11(2-3):164-170.

6Boyd A, Chakrabarty AM. Pseudomonas aeruginosa biofilms: role of the alginate exopolysaccharide. J of Industrial Microbiology. 1995;15:162-8.

7Hunt BE, Weber A, Berger A, Ramsey B, Smith AL. Macromolecular mechanisms of sputum inhibition of tobramycin activity. Antimicrobial Agents and Chemotherapy. 1995;39(1):34-9.

8Nichols WW, Dorrington SM, Slack MP, Walmsley HL. Inhibition of tobramycin diffusion by binding to alginate. Antimicrobial Agents and Chemotherapy. 1988;32(4):518-23.

9Gordon CA, Hodges NA, Marriott C. Antibiotic interaction and diffusion through alginate and exopolysaccharide of cystic fibrosis-derived Pseudomonas aeruginosa. J Antimicrob Chemother. 1988;22(5):667-74.