Of the 68 patients who completed the 84-day open label trial, 21 were culture negative at day 168 (the open label segment followed an 84-day double blind trial). Of the 21 culture negative patients, 10 were culture negative at the beginning of the open label segment. The other eleven culture negatives were five patients in the Arikayce arm and six patients who switched from placebo to Arikayce. This implies a cure rate of ~16%.
In the double blind segment of the trial, 11 of 44 Arikayce patients were culture negative by day 84 implying a cure rate of 25%.
In April, the firm submitted its application to the FDA for BTD designation for Arikayce. It is already designated an Orphan Drug, a Qualified Infectious Disease Product and Fast Track for the treatment of nontuberculous mycobacterial lung infections.