FDA Accepts Insmed’s Application to Market ALIS for Rare Lung Disease

Rare Daily Staff

The U.S. Food and Drug Administration has accepted Insmed’s application to market ALIS (Amikacin Liposome Inhalation Suspension) for adult patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex.

NTM lung disease is a rare and serious disorder associated with increased rates of morbidity and mortality. There is an increasing prevalence of lung disease caused by NTM. Patients with NTM lung disease may experience a multitude of symptoms such as fever, weight loss, cough, lack of appetite, night sweats, blood in the sputum, and fatigue. Patients with NTM lung disease frequently require lengthy hospital stays to manage their condition.

ALIS is a novel, inhaled, once-daily formulation of amikacin. Though Amikacin solution for parenteral administration is an established drug that has activity against a variety of NTM, its use is limited by the need to administer it intravenously and by toxicity to hearing, balance, and kidney function. Insmed’s advanced pulmonary liposome technology uses charge neutral liposomes to deliver amikacin directly to the lung where it is taken up by the lung macrophages where the NTM infection resides. This prolongs the release of amikacin in the lungs while minimizing systemic exposure thereby offering the potential for decreased systemic toxicities.

The FDA granted Insmed’s request for Priority Review and has set an action date of September 28, 2018 under the Prescription Drug User Fee Act. FDA has previously designated ALIS as an orphan drug, a breakthrough therapy, and a Qualified Infectious Disease Product GAIN Act.

Insmed expects that the FDA will convene an advisory committee meeting to discuss this application.

May 17, 2017