FDA Advisory Committee Votes in Favor of ALIS to Treat NTM Lung Disease Caused by MAC

The US Food and Drug Administration (FDA)’s Antimicrobial Drugs Advisory Committee has voted (12 to 2) in favor of the safety and effectiveness of Insmed Incorporated’s amikacin liposome inhalation suspension (ALIS) for the treatment of adults with nontuberculosis mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC) who have limited or no treatment options available.

If approved, ALIS will be the first and only therapy available in the United States that will be specifically indicated for the treatment of patients with NTM lung disease caused by MAC.

The committee also voted in favor of the surrogate endpoint of sputum culture conversion used in the phase 3 CONVERT trial as being reasonably likely to predict clinical benefit, according to the announcement.

“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 need among patients suffering from NTM lung disease caused by MAC, a chronic, debilitating and potentially fatal infection,” Will Lewis, Insmed’s president and chief executive officer, said in a recent statement.

The committee based their recommendations off of materials developed from the pharmaceutical company’s new drug application (NDA), which had been submitted to the FDA under accelerated approval provisions; the NDA includes data gleaned from the pivotal phase 3 CONVERT trial.

For the randomized, open-label phase 3 trial, investigators sought to confirm the positive results yielded from a phase 2 trial completed in 2014. The trial enrolled 336 patients and found that adding ALIS to standard therapy for NTM lung infections caused by MAC resulted in no evidence of NTM lung disease at month 6 for 29% of patients compared to 9% of patients on standard therapy alone.

The patients enrolled in the phase 3 trial are representative of a portion of the NTM lung disease population that are most difficult-to-treat, according to Paul Streck, MD, Insmed’s chief medical officer; these patients have already failed treatment with current guideline-based therapy.

In a separate poll, the committee decided to vote against the safety and effectiveness of the therapy for the broadest population of adult patients with NTM lung disease caused by MAC.

The FDA will take the committee’s recommendation into consideration when it comes time to decide whether to approve Insmed’s NDA for ALIS, which is currently under Priority Review. September 28, 2018 has been identified as the action date for this decision.

Previous to this vote, ALIS has received orphan drug and breakthrough therapy designations from the FDA. ALIS has also been designated as a Qualified Infectious Disease Product under the Generating Antibiotic Incentives Now Act.