The FDA’s Antimicrobial Drugs Advisory Committee (AMDAC) voted against the efficacy and safety of Insmed’s ALIS amikacin liposome inhalation suspension for the treatment of nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC) for all patients but voted in favor of its efficacy and safety for patients with limited or no other treatment options. Insmed submitted the NDA for ALIS in March 2018.
The vote was 2 yes, 12 no on the question “Has the applicant provided substantial evidence of the effectiveness and sufficient evidence of the safety of amikacin liposomal inhalation solution (ALIS) for the treatment of nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex as part of a combination antibacterial drug regimen for adult patients?”
However, on the question, “Has the applicant provided substantial evidence of the effectiveness and sufficient evidence of the safety of ALIS for the treatment of nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex as part of a combination antibacterial drug regimen for adult patients with limited or no treatment options?”, the vote was 12 yes, 2
no.
The committee also addressed a question relevant to a surrogate endpoint used in the Phase 3 CONVERT study: “Is the surrogate endpoint of sputum culture conversion based on three consecutive negative sputum cultures reasonably likely to predict clinical benefit?” On that question, the vote was 8 yes, 6 no.
Insmed President and CEO Will Lewis said, “We are very pleased by the outcome of today’s advisory committee meeting, which recognized the role ALIS may be able to play in addressing the significant unmet medical need among patients suffering from NTM lung disease caused by MAC, a chronic, debilitating and potentially fatal infection.”
Chief Medical Officer Paul Streck said, “The patients included in our Phase 3 clinical trial represent the most difficult-to-treat segment of the NTM lung disease population, having already failed treatment with current guideline-based therapy. The committee’s favorable recommendation brings us one step closer to providing the first and only FDA-approved treatment for these patients. We look forward to working closely with the FDA as it completes its review of our application.”
Read the Insmed press release.
