FDA Approves New Drug for Serious Lung Disease

First Drug Approved Under Agency’s LPAD Pathway

The FDA has approved a new drug, Arikayce (amikacin liposome inhalation suspension, Insmed, Inc., Bridgewater, NJ ), to treat lung disease caused by certain bacteria (MAC) in patients who do not respond to conventional treatment.

Patients with MAC (Mycobacterium avium complex), often found in water and soil, can suffer from various symptoms including persistent cough, fatigue, weight loss, night sweats, breathlessness, and coughing up blood. After six months of treatment with Arikayce, 29% of patients showed no increase of mycobacteria for 90 days, compared to only 9% of those who had not received treatment.

Due to the increasing resistance of bacteria to existing antibiotics, FDA Commissioner Scott Gottlieb, MD, stressed the importance of using novel methods for R&D to encourage investment. The approval of Arikayce, under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD), represents an important policy milestone. The pathway, advanced by Congress, aims to increase the development of drugs to tackle infections lacking effective therapies.

In addition to being approved under the Accelerated Approval pathway, Arikayce has received Fast Track, Breakthrough Therapy, Priority Review, and Qualified Infectious Disease Product (QIDP) designations. Such designations are given to antibacterial products that treat serious or life-threatening infections under the Generating Antibiotic Incentives Now (GAIN) title. Arikayce also received Orphan Drug designation, which encourages the development of drugs for rare diseases.

Source: FDA.gov, October 1, 2018