WASHINGTON -- The FDA approved Insmed's amikacin liposome inhalation suspension (Arikayce) for a limited population of patients with a certain type of non-tuberculosis mycobacterial lung infection, the agency said today.
In what was described as the first drug granted approval under FDA's Limited Population Pathway for Antibacterial and Antifungal Drugs, Arikayce for treatment of lung disease caused by Mycobacterium avium complex (MAC) for patients who do not respond to conventional treatment.
The was established under the , and FDA commissioner Scott Gottlieb, MD, discussed the need for development of drugs to treat resistant infections.
"This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies," he said in a statement. "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."
The FDA also noted that the drug was approved under the , which is reserved for 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."
An FDA advisory committee meeting in August voted to recommend approval of the treatment as part of a combination antibacterial regimen in patients with limited or no treatment options. As part of the accelerated approval process, safety and efficacy of the drug was based on trials that were conducted using a surrogate endpoint -- three successive negative sputum cultures. The committee also voted against recommending the therapy among all patients with this type of lung disease.
Arikayce will have a Boxed Warning about increased risk of respiratory conditions, such as hypersensitivity pneumonitis, bronchospasm, exacerbation of underlying lung disease and hemoptysis, the agency said.