The FDA’s Antimicrobial Drugs Advisory Committee voted 12 to 2 in favor of the safety and effectiveness of ALIS for adults with 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 adults with nontuberculosis mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC) who have limited or no treatment options.
If approved, this treatment will be the first and only therapy that is specifically indicated for the treatment of patients with NTM lung disease caused by MAC in the United States.
The committee also voted in favor of the surrogate endpoint of sputum culture conversion used in the Phase 3 CONVERT study 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 medical need among patients suffering from NTM lung disease caused by MAC, a chronic, debilitating, and potentially fatal infection,” Insmed Incorporated’s president and chief executive officer Will Lewis said in a recent statement.
The committee based their recommendation on materials developed from the pharmaceutical’s new drug application for the therapy, which was submitted under accelerated approval provisions. The application includes data yielded from the phase 3 CONVERT study.
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 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,” Paul Streck, MD, Insmed’s chief medical officer, added. “The committee’s favorable recommendation brings us one step closer to providing the first and only FDA-approved treatment for these patients.”
However, according to the announcement, the committee also voted against the safety and effectiveness of ALIS in the broadest population of adult patients with NTM lung disease caused by MAC.
The FDA will take the committee’s recommendation into consideration when deciding whether to approve the NDA for ALIS which is currently under Priority Review. The FDA is estimated to make their decision on September 28, 2018.
Previous to this, ALIS has been granted orphan drug and breakthrough therapy designations. This drug was also designated as a Qualified Infectious Disease Product under the Generating Antibiotics Incentives Now Act.
NTM lung disease is a rare and serious disorder that is known to have high rates of morbidity and mortality. Patients with this rare disorder are known to experience several symptoms including fever, weight loss, a cough, loss of appetite, night sweats, blood in the sputum, and fatigue.