FDA Approves Antibacterial Treatment for Lung Disease Through Novel Pathway


The FDA has approved amikacin liposome inhalation suspension (Arikayce, Insmed), for the treatment of lung disease caused by a group of bacteria in a limited population of patients with the disease who do not respond to conventional treatment.

The FDA has approved amikacin liposome inhalation suspension (Arikayce, Insmed), for the treatment of lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC) in a limited population of patients with the disease who do not respond to conventional treatment.

MAC is a type of nontuberculous mycobacteria (NTM) that is commonly found in water and soil. Symptoms of MAC include persistent cough, fatigue, weight loss, night sweats, and occasionally shortness of breath and coughing up of blood.

According to FDA Commissioner Scott Gottlieb, MD, the agency’s approval of amikacin liposome inhalation suspension marks an important policy milestone. It is the first time a drug is approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs.

"This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies. We’re seeing a lot of early interest among sponsors in using this new pathway, and it’s our hope that it’ll spur more development and approval of antibacterial drugs for treating serious or life-threatening infections in limited populations of patients with unmet medical needs," said Gottlieb, in a prepared statement. "As bacteria continue to grow impervious to currently available antibiotics, we need to encourage the development of drugs that can treat resistant infections. That means utilizing novel tools intended to streamline development and encourage investment into these important endeavors."

Arikayce also was approved under the Accelerated Approval pathway. Under this approach, the FDA may approve drugs for serious or life-threatening diseases or conditions where the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. The approval of this amikacin liposome inhalation suspension was based on achieving 3 consecutive negative monthly sputum cultures by month six of treatment. The sponsor of Arikayce will be required by the FDA to conduct an additional, post-market study to describe the clinical benefits of the drug.

The safety and efficacy of amikacin liposome inhalation suspension, a treatment taken through a nebulizer, was demonstrated in a randomized, controlled clinical trial where patients were assigned to 1 of 2 treatment groups. One group of patients received amikacin liposome inhalation suspension plus a background multidrug antibacterial regimen, while the other treatment group received a background multidrug antibacterial regimen alone. By the sixth month of treatment, 29% of patients treated with amikacin liposome inhalation suspension had no growth of mycobacteria in their sputum cultures for 3 consecutive months compared to 9% of patients who were not treated with Arikayce.

The drug’s prescribing information includes a Boxed Warning regarding the increased risk of respiratory conditions including inflamed lungs, tightening of the airway, exacerbation of underlying lung disease and spitting up blood that have led to hospitalizations in some cases. Other common adverse effects in patients taking Arikayce were difficulty speaking, cough, damaged hearing, upper airway irritation, musculoskeletal pain, fatigue, diarrhea and nausea.

The FDA granted this application Fast Track, Breakthrough Therapy, Priority Review, and Qualified Infectious Disease Product (QIDP) designations. QIDP designation is given to antibacterial products that treat serious or life-threatening infections under the Generating Antibiotic Incentives Now (GAIN) title of the FDA Safety and Innovation Act. Arikayce also received Orphan Drug designation, which provides additional incentives to assist and encourage the development of drugs for rare diseases.


FDA approves a new antibacterial drug to treat a serious lung disease using a novel pathway to spur innovation [news release]. Silver Spring, MD; September 28, 2018: FDA website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm622048.htm. Accessed September 28, 2018.

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