The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended Arikayce — liposomal amikacin for inhalation — as a therapy for lung infections caused by Mycobacterium avium complex (MAC), a type of non-tuberculous mycobacteria (NTM), in adults without cystic fibrosis and with limited treatment options.
The European Commission now will review the CHMP opinion, with a final decision expected by the end of this year.
Arikayce, given at 590 mg in a nebulizer, previously received orphan drug designation for the treatment of NTM lung infections in the European Union. But in 2016, Insmed — the company developing Arikayce — decided to withdraw a first application seeking approval of the treatment in the EU; Insmed did so because the CHMP had issued a provisional opinion that Arikayce’s benefits did not outweigh its risks in people with MAC lung infection.
As part of a combination regimen, Arikayce became the first therapy approved by the U.S. Food and Drug Administration for the treatment of adults with resistant MAC infections. Insmed is awaiting a regulatory decision in Japan on whether to approve the therapy for the same indication.
“Today’s positive opinion from the CHMP marks an important step in our journey to transform the way MAC lung disease is managed for patients around the world. If approved by the EC, ARIKAYCE would be the first therapy in both the European Union and the United States for patients with this chronic, debilitating condition,” Will Lewis, Insmed chairman and CEO, said in a press release.
“We look forward to potentially bringing ARIKAYCE to appropriate patients in Europe as quickly as possible,” he said.
Arikayce’s active ingredient, the antibiotic amikacin, kills bacteria by preventing their production of proteins they need for survival. Specifically, amikacin is effective against gram-negative bacteria and mycobacteria, which can lead to severe lung complications in people with bronchiectasis.
The medication is delivered in liposomes, which are water-filled, tiny vesicles made of the same material (phospholipids) as the cell membrane. Without liposomes, amikacin may be trapped by a substance produced by bacteria before the therapy reaches immune cells called macrophages in the lung.
The CHMP based its opinion on the results of the Phase 3 CONVERT clinical trial (NCT02344004), which recruited 336 participants with refractory NTM lung disease caused by MAC. Results demonstrated that once-daily Arikayce, taken in combination with a standard multi-drug regimen (MDR), cleared MAC from sputum more effectively than standard MDR therapy alone.
“The positive CHMP opinion underscores the potential benefit of ARIKAYCE in the management of MAC lung disease for patients who are particularly difficult to treat and continue to suffer in the absence of an approved therapy in Europe,” said Christoph Lange, medical director of clinical infectious diseases at the Leibniz Lung Center, in Germany.
Leading international respiratory medicine and infectious disease societies recently issued new treatment guidelines, recommending Arikayce in combination with an MDR for the treating patients whose MAC lung disease had resisted standard therapies for at least six months.