Arikayce (liposomal amikacin for inhalation, or LAI), by Insmed, is approved to treat adults with few or no treatment options for lung infections caused by Mycobacterium avium complex (MAC), a type of non-tuberculous mycobacteria (NTM)

Arikayce consists of liposomes that contain amikacin, an antibiotic used to treat severe or serious bacterial infections.

How does Arikayce work?

The active ingredient of Arikayce is amikacin, an aminoglycoside antibiotic that works by interrupting bacterial protein synthesis. Amikacin can treat infections caused by aerobic, gram-negative bacteria and mycobacteria, the bacteria that cause tuberculosis, which can lead to serious lung complications in people with bronchiectasis.

Inhaled amikacin is typically inefficient in treating MAC infections, as the bacteria can produce a negatively charged substance that attracts and traps the positively charged amikacin before it can reach the pathogen.

Arikayce uses liposomes to bypass this trap. Liposomes are microscopic, phospholipid spheres containing water. In Arikayce, the water-soluble amikacin can be contained at high concentrations inside the liposome. Insmed’s liposomal technology is designed to deliver amikacin directly to the site of infection in the lungs for immediate treatment, and to maintain the antibiotic there over time.

MAC has come to represent a high risk in terms of its incidence, prevalence, hospitalization rate, and mortality rate. People with chronic pulmonary conditions like non-cystic fibrosis bronchiectasis are particularly susceptible. MAC infection symptoms often overlap with those of the underlying disease, so infections can go undiagnosed until later stages. 

Arikayce in clinical trials

A Phase 2 study (NCT01315236) assessed the effectiveness, safety, and tolerability of Arikayce, versus placebo, in about 90 people with chronic NTM infections who failed to respond to standard antibiotics. Patients received 590 mg of the inhalation therapy, once daily, for 84 days. Results showed that Arikayce efficiently people with MAC infection, but was not superior to placebo for patients with Mycobacterium abcessus infection.

Fifty-nine patients who completed this trial moved into its open-label extension study, being treated with Arikayce for 84 days plus standard care (an anti-mycobacterial regimen). Results at one year of monitoring supported those of the Phase 2 study. Participants who achieved negative sputum cultures continued to be free of infection, and those given Arikayce in the main study as well as its extension showed a continued improvement in the six-minute walk test, a measure of lung health. Those who switched from placebo to treatment in the extension showed a slowing rate of decline.

An open-label Phase 3 study (NCT02344004), called CONVERT, evaluated Arikayce plus standard treatment versus standard treatment alone in 336 patients with resistant MAC infections. Study results at six months, published in the American Journal of Respiratory and Critical Care Medicine, showed that 29% of patients on Arikayce achieved culture conversion, compared with 8.9% of those on standard care. Culture conversion was defined as three consecutive months of sputum cultures negative for the presence of MAC.

The U.S. Food and Drug Administration approved Arikayce for the specific treatment of adults with resistant MAC infections in September 2018, based on CONVERT trial findings.

A Phase 2 open-label and investigator-initiated study (NCT03038178) tested the safety, effectiveness, and tolerability of Arikayce, taken once daily with standard therapy for 12 months, in treating a different strain of bacteria, Mycobacterium abcessus. Its primary goal was culture conversion in sputum cultures. Secondary goals included changes in the six-minute walk test with treatment and reported adverse events. Insmed presented interim results on 33 patients in May 2019, showing that 37%  had achieved stable culture conversion after 12 months. The study end in December 2019; further results are not yet public.

Additional information

The European Union approved Arikayce for adults with NTM lung disease due to MAC who fail to respond sufficiently to other therapies in October 2020. The treatment is under review in Japan.


Last updated: Feb. 5, 2021


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