Insmed announced that the Food and Drug Administration (FDA)’s Antimicrobial Drugs Advisory Committee voted in favor (12-2) for the safety and efficacy of ALIS (amikacin liposome inhalation suspension) for adults with nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC) who have limited or no treatment options.
The Advisory Committee also voted in favor that the surrogate endpoint of sputum culture conversion used in the Phase 3 CONVERT study is reasonably likely to predict clinical benefit. In the CONVERT study, the primary endpoint of culture conversion by month 6 was met with statistical significance in favor of once-daily ALIS with guideline-based therapy (GBT) vs GBT alone in patients with refractory NTM lung disease due to MAC.
The Committee, however, voted against the safety and efficacy of ALIS for the broadest population of adults with NTM lung disease caused by MAC.
ALIS is a novel formulation of amikacin, an antibiotic with established activity against a variety of NTM. The pulmonary liposome technology allows neutral liposomes to deliver amikacin directly to the lung where it is taken up by the lung macrophages to the site of infection. This prolongs amikacin release in the lungs while minimizing systemic exposure. ALIS would be given as a once-daily inhalation using the eFlow Nebulizer System.
ALIS would be the first treatment in the US for patients with NTM lung disease caused by MAC, if approved. The FDA is not bound by the Committee’s recommendations but takes them into consideration for approval. The Prescription Drug User Fee Act (PDUFA) target date has been set for September 28, 2018.
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