Arikayce (also known as liposomal amikacin for inhalation, or LAI) is an approved treatment for adults developed by Insmed to treat lung infections caused by Mycobacterium avium complex (MAC), a type of non-tuberculous mycobacteria (NTM), which have not responded to standard treatments. Arikayce consists of liposomes that contain amikacin, an antibiotic used to treat severe or serious bacterial infections.

Arikayce was approved for use in the U.S. in September 2018 and the European Union in October 2020. It is also under review in Japan.

How Arikayce works

The active ingredient of Arikayce is amikacin. This is an aminoglycoside antibiotic that works by interrupting bacterial protein synthesis after binding to the bacteria. Amikacin is used to 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.

Normal inhaled amikacin can be inefficient in treating MAC infections, as the bacteria can produce a negatively-charged substance that can attract and trap the positively charged amikacin before it can reach the bacteria.

Arikayce uses liposomes to bypass this. Liposomes are microscopic phospholipid spheres that contain water, and 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 locally in the lungs over time.

In recent years, MAC has come to represent a high risk in terms of its incidence, prevalence, hospitalization rate, and mortality rate, and people with chronic pulmonary conditions like bronchiectasis are particularly susceptible. MAC infection symptoms often overlap with those of the underlying disease, so that MAC infections aren’t often diagnosed until a later stage. Treatments that might address this disease, especially at earlier stages, are of growing interest.

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. Treated patients were given 590 mg of the inhalation therapy, once daily, for 84 days. Trial results showed that Arikayce is efficient in treating people with Mycobacterium avium complex infection, but that the treatment was not superior to placebo for those patients with Mycobacterium abcessus infection.

Participants completing the Phase 2 trial had the option of continuing Arikayce in an open-label extension study. The 59 patients who completed the extension study received Arikayce for 84-days plus standard care (an anti-mycobacterial regimen), and were monitored for 12 months. The results supported those of the Phase 2 study. Participants who achieved negative sputum cultures continued to be free of infection in both the open-label and follow-up phases. Patients who were assigned Arikayce in the Phase 2 study showed a continued improvement in the six-minute walk test, while those who switched from placebo to treatment in the open-label trial demonstrated a reduced rate of decline from baseline.

An open-label Phase 3 study (NCT02344004), called CONVERT, evaluated Arikayce with standard treatment versus standard treatment alone in 336 patients with resistant MAC infections. Patients were randomized 2:1 between the Arikayce and standard of care alone arms of the trial. Results of the study, published in the American Journal of Respiratory and Critical Care Medicine, showed that, after 6 months of treatment, 29% of patients receiving Arikayce achieved culture conversion compared to just 8.9% of patients receiving standard care.  Culture conversion was achieved when patients had 3 consecutive months of sputum (a mixture of spit and mucus used to test for lung infection) cultures that tested negative for the presence of MAC.

Based on the findings from the CONVERT trial, Insmed received FDA approval for Arikayce in the specific treatment of adults with resistant MAC infections in September 2018.

A Phase 2 open-label extension study (NCT03038178) aims to test the safety, effectiveness, and tolerability of Arikayce taken once daily along with standard therapy for 12 months in the treatment of a different strain of bacteria, Mycobacterium abcessus. The primary goal of the study is culture conversion in sputum cultures. Secondary goals will be to record changes in the six-minute walk test and adverse events. Insmed presented interim results of 33 participants from the study in May of 2019 showing that 37% of patients had achieved stable culture conversion after 12 months. The study completed in October of 2019 and final results are awaited.


Last updated: Dec. 27, 2020


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