The European Commission has approved Arikayce — liposomal amikacin for inhalation — as the first treatment for lung infections caused by Mycobacterium avium complex (MAC), a type of non-tuberculous mycobacteria (NTM), in adults without cystic fibrosis (CF) and with limited treatment options.
“We are thrilled that for the first time, non-CF patients with NTM lung infections caused by MAC in the European Union (EU) have an approved therapy to help manage this difficult-to-treat condition, providing a new approach for those who have suffered with few, or no, treatment options,” Will Lewis, the chairman and CEO of Insmed, the therapy’s developer, said in a press release.
“Today’s approval underscores our commitment to serving the MAC lung disease community around the world, and we look forward to bringing ARIKAYCE to appropriate patients in the EU,” Lewis added.
Insmed plans to launch Arikayce first in Germany, followed by the U.K. and other EU markets, subject to decisions on public reimbursement by health authorities in each European Union member state.
The company also has established country-specific programs to provide patients with ongoing support and information regarding Arikayce.
The approval was supported by a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) — a branch of the European Medicines Agency — which in 2016 had issued a provisional opinion against approving Insmed’s first marketing applications for Arikayce.
Predicting that the European Commission would not approve the therapy based on the then-available Phase 2 trial data, Insmed withdrew its application, planning to resubmit it when clinical data from the global Phase 3 CONVERT study (NCT02344004) were available.
With this approval, Arikayce becomes the first and only therapy approved in both the EU and the U.S. for MAC lung disease. In the U.S., the therapy is approved, as part of a combination regimen, for adults with resistant MAC infections who have limited or no alternative treatment options. Arikayce also is under regulatory review in Japan for a similar indication.
Arikayce is an inhaled antibiotic taken once a day with a nebulizer at a dose of 590 mg. Its active ingredient, amikacin, kills bacteria by preventing its production of proteins it needs to survive.
Specifically, amikacin is effective against gram-negative bacteria and mycobacteria, which can cause severe lung manifestations in people with bronchiectasis.
Arikayce differs from other inhaled antibiotics as it delivers amikacin in liposomes — water-filled, tiny vesicles made of the material (phospholipids) found in cell membranes.
These liposomes are designed to help amikacin reach the lungs, as without them, the therapy may be trapped by a substance produced by bacteria.
Arikayce’s approvals were based on positive results from the CONVERT trial, which involved 336 participants with treatment-resistant NTM lung disease caused by MAC.
The results showed that Arikayce, taken in combination with a standard multi-therapy regimen, cleared MAC from patients’ sputum more effectively than standard treatment alone. Arikayce’s most common side effects affecting the respiratory system were hoarseness, cough, and shortness of breath.
“The treatment of MAC lung disease is challenging, with a significant need for new therapies that improve upon the current standard of care and offer options to patients who previously have not been treated successfully,” said Marc Lipman, MD, a professor of respiratory medicine at University College London and one of the founding trustees of NTM Patient Care UK.
Michael Loebinger, PhD, an investigator in CONVERT, said that the “results from the landmark CONVERT study show that adding Arikayce has the potential to help patients who were [resistant] to standard treatment achieve [MAC clearance]—a critical outcome.”
“Today’s approval marks a significant milestone in advancing care for patients with MAC lung disease in the EU,” added Loebinger, also a respiratory consultant at Royal Brompton Hospital and a professor of practice (respiratory medicine) at Imperial College London.
CHMP also adopted a positive opinion recommending that Arikayce should maintain its orphan drug designation in the EU for the treatment of NTM lung disease, which was originally granted in 2014. This designation provides regulatory support and financial benefits, as well as 10 years of marketing exclusivity upon regulatory approval.