FDA Approves Amikacin Liposome Inhalation Suspension for Lung Disease Caused by MAC Bacteria

The US Food and Drug Administration (FDA) has approved amikacin liposome inhalation suspension (Arikayce) for the treatment of lung disease caused by Mycobacterium avium complex (MAC) in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease).

Amikacin liposome inhalation suspension was approved under accelerated approval.

"This approval is the first time a drug is being approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, and it marks an important policy milestone,” said FDA Commissioner Scott Gottlieb, M.D., in a recent statement. “This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies.”

A streamlined clinical development program may support approval under the LPAD pathway, which may involve smaller, shorter or fewer clinical trials.

Achievement of 3 consecutive negative monthly sputum cultures by month 6 of treatment served as the basis for the treatment. An additional, post-market study will be required by the drug’s sponsor in order to describe its clinical benefits.

The safety and efficacy of the inhaled treatment taken through a nebulizer was shown in a randomized, controlled clinical trial. Participating patients were divided to 1 of 2 treatment groups.

Patients in 1 group were administered amikacin liposome inhalation suspension in addition to a background multi-drug antibacterial regimen, while patients in another group were administered a background multi-drug antibacterial regimen alone.

In patients treated with the drug, 29% had no growth of mycobacteria in their sputum cultures by the 6th month for 3 consecutive months compared to 9% of patients who were not treated with the inhalation suspension.

The risk of respiratory conditions including hypersensitivity pneumonitis (inflamed lungs), bronchospasm (tightening of the airway), exacerbation of underlying lung disease, and hemoptysis (spitting up blood) that have led to hospitalizations in some cases are Boxed Warnings with amikacin liposome inhalation.

Dysphonia (difficulty speaking), cough, ototoxicity (damaged hearing), upper airway irritation, musculoskeletal pain, fatigue, diarrhea, and nausea include other common side effects with the drug.

Previously, amikacin liposome inhalation suspension was granted a fast track, breakthrough therapy, priority review, and qualified infectious disease product (QIDP) designation.