FDA grants accelerated approval to Arikayce to treat mycobacterium avium complex lung disease.-Insmed

Insmed Incorporated announced that the FDA has granted accelerated approval of Arikayce (amikacin liposome inhalation suspension) for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options. Arikayce is the first and only therapy approved in the U.S. specifically for patients with MAC lung disease, a chronic and debilitating condition that can significantly increase patient morbidity and mortality.

Arikayce is the first product approved via the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD). LPAD, which was enacted as part of the 21st Century Cures Act, serves to advance the development of new antibacterial drugs to treat serious or life-threatening infections in limited populations of patients with unmet needs.

The approval of Arikayce under FDA�s LPAD and accelerated approval pathways was based on results from the ongoing Phase III CONVERT study, which has demonstrated that Arikayce , when combined with guideline-based therapy (GBT), improved sputum culture conversion rates. The global CONVERT study met its primary endpoint of sputum culture conversion by Month 6 with statistical significance for once-daily Arikayce when added to GBT compared with GBT alone (p<0.0001) in patients with refractory nontuberculous mycobacterial ntm lung disease caused by mac. in the study the addition of arikayce to gbt eliminated evidence of ntm lung disease caused by mac in sputum by month 6 in 29 of patients compared to 9 of patients on gbt alone.>

As a condition of accelerated approval, Insmed is collaborating with the FDA on the design of an additional clinical study to support full approval. The study design is currently under discussion with FDA and is proposed to be a randomized, double-blind, placebo-controlled clinical trial to assess and describe the clinical benefit of Arikayce in patients with NTM lung disease caused by MAC. The trial will evaluate the effect of Arikayce on a clinically meaningful endpoint, as compared to an appropriate control, in the intended patient population of patients with MAC infection. Insmed will provide additional updates once the study design has been finalized with FDA. Continued approval of Arikayce will be contingent upon verification and description of clinical benefit in this study.