
FDA Approves New Drug for Bacterial Lung Disease
Insmed’s Arikayce (amikacin liposome inhalation suspension) is the first drug approved under the agency’s Limited Population Pathway for Antibacterial and Antifungal Drugs, established to support development of antibiotics for unmet medical needs.
On Sept. 28, 2018, FDA announced the approval of Arikayce (amikacin liposome inhalation suspension) for the treatment of lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC), in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease).
MAC is a type of nontuberculous mycobacteria (NTM) found in water and soil. Symptoms of disease in patients with MAC include persistent cough, fatigue, weight loss, night sweats, and occasional shortness of breath and coughing up of blood.
"As bacteria continue to grow impervious to currently available antibiotics, we need to encourage the development of drugs that can treat resistant infections. That means utilizing novel tools intended to streamline development and encourage investment into these important endeavors," said FDA Commissioner Scott Gottlieb, MD, in an agency press release.
Arikayce is an inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. FDA granted approval of the drug to Insmed, a Bridgewater, NJ-based biopharmaceutical company focused on rare diseases.
Insmed’s proprietary Pulmovance liposomal technology enables the delivery of amikacin directly to the lungs, where it is taken up by lung macrophages where the infection resides. This approach prolongs the release of amikacin in the lungs while limiting systemic exposure, according to Insmed. The drug is administered once daily using the Lamira Nebulizer System, manufactured by PARI Pharma. Lamira is a quiet, portable nebulizer that enables efficient aerosolization of liquid medications, including liposomal formulations, via a vibrating, perforated membrane.
FDA reports that Arikayce is the first drug to be approved under the
Arikayce also was approved under the
FDA granted this application fast track, breakthrough therapy, priority review, and qualified infectious disease product (QIDP) designations. QIDP designation is given to antibacterial products that treat serious or life-threatening infections under the Generating Antibiotic Incentives Now (GAIN) title of the FDA Safety and Innovation Act. Arikayce also received orphan drug designation, which provides additional incentives to assist and encourage the development of drugs for rare diseases.
"This approval is the first time a drug is being approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, and it marks an important policy milestone. This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies,” Gottlieb stated in the release. “We’re seeing a lot of early interest among sponsors in using this new pathway, and it’s our hope that it’ll spur more development and approval of antibacterial drugs for treating serious or life-threatening infections in limited populations of patients with unmet medical needs."
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