that has been caused by members of the Mycobacterium avium complex and is refractory to other treatments.
In a randomized, controlled trial, patients with refractory M. avium complex infections were assigned to receive either Arikayce plus a multidrug antibacterial regimen or just the antibacterial regimen. By 6 months, sputum cultures for 29% of those treated with the combination had shown no mycobacterial growth for 3 consecutive months, whereas this was only true for the cultures for 9% of patients on the multidrug antibacterial regimen alone.
The Arikayce prescribing information includes a boxed warning regarding the increased risk of respiratory conditions, including hypersensitivity pneumonitis, bronchospasm, exacerbation of underlying lung disease, and hemoptysis, some of which have proven serious enough to lead to hospitalization. Other side effects include dysphonia, cough, musculoskeletal pain, nausea, and fatigue.
According to the press announcement from the FDA, this is the first approval under the Limited Population Pathway for Antibacterial and Antifungal Drugs, which was set up by Congress “to advance development and approval of antibacterial and antifungal drugs to treat serious or life-threatening infections in a limited population of patients with unmet need.” It does so by allowing a more streamlined clinical development program that may involve smaller, shorter, or fewer clinical trials.
More information can be found in the full press announcement.