Amikacin Liposome Inhalation Suspension: A Review in Mycobacterium avium Complex Lung Disease
Amikacin liposome inhalation suspension (ALIS; Arikayce®) [formerly known as liposomal amikacin for inhalation, or LAI] is a liposomal formulation of the aminoglycoside antibacterial drug amikacin. The ALIS formulation, administered via inhalation following nebulization, is designed to facilitate targeted and localized drug delivery to the lungs while minimizing systemic exposure. Based on the prespecified primary endpoint analysis of the ongoing phase III CONVERT trial, ALIS has been approved in the USA for use as part of a combination antibacterial drug regimen against Mycobacterium avium complex (MAC) lung disease that is treatment refractory (i.e. an active infection present despite ≥ 6 consecutive months of a multidrug regimen) in adult patients who have limited or no alternative treatment options. In the CONVERT trial, once-daily ALIS as an add-on to guidelines-based therapy (GBT) significantly increased the odds of achieving sputum culture conversion by month 6 compared with GBT alone in patients with treatment-refractory MAC lung disease. The addition of ALIS to GBT was associated with an increased risk of respiratory adverse events compared with GBT alone; however, serious adverse events were experienced by a similar proportion of patients in the two treatment groups. In conclusion, although current evidence for efficacy is limited to microbiological outcomes (with clinical benefit yet to be established), available data suggest that ALIS is a useful option for the treatment of patients with MAC lung disease who have not responded to conventional therapy and for whom there are limited or no alternative treatment options available.
During the peer review process, the manufacturer of ALIS was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.
Compliance with Ethical Standards
The preparation of this review was not supported by any external funding.
Conflict of interest
Matt Shirley is a salaried employee of Adis/Springer, is responsible for the article content and declares no relevant conflicts of interest.
- 7.Winthrop K, Adjemian J, Mirsaeidi M, et al. Incidence and prevalence of nontuberculous mycobacterial lung disease in US Medicare beneficiaries, 2008–2015 [abstract 780 and poster]. In: ID week. 2018.Google Scholar
- 14.US FDA. Arikayce® (amikacin liposome inhalation suspension): US prescribing information. 2018. http://www.accessdata.fda.gov/drugsatfda_docs/label/2018/207356s000lbl.pdf. Accessed 19 Feb 2019.
- 15.National Institutes of Health—US National Library of Medicine. Amikacin sulfate injection: US prescribing information. 2018. http://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6ec3129b-c53b-4bdb-913d-a2d0060fa140. Accessed 19 Feb 2019.
- 22.Insmed Ltd. Amikacin liposome inhalation suspension: Antimicrobial Drugs Advisory Committee briefing materials. 2018. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/Anti-InfectiveDrugsAdvisoryCommittee/UCM615723.pdf. Accessed 19 Feb 2019.
- 23.Olivier KN, Maass-Moreno R, Whatley M, et al. Airway deposition and retention of liposomal amikacin for inhalation in patients with pulmonary nontuberculous mycobacterial disease [abstract A3732 and poster]. Am J Respir Crit Care Med. 2016;193:A3732.Google Scholar
- 24.Brown-Elliott BA, Eagle G, Wallace Jr RJ, et al. Amikacin liposome inhalation suspension (ALIS) add-on therapy for refractory Mycobacterium avium complex (MAC) lung disease: effect of in vitro amikacin susceptibility on sputum culture conversion [abstract 805 and poster]. In: ID week. 2018.Google Scholar