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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 Limited Population Pathway for Antibacterial and Antifungal Drugs, or LPAD pathway, established by Congress under the 21st Century Cures Act to advance development and approval of antibacterial and antifungal drugs to treat serious or life-threatening infections in a limited population of patients with unmet need. Approval under the LPAD pathway may be supported by a streamlined clinical development program. These programs may involve smaller, shorter, or fewer clinical trials, according to FDA. As required for drugs approved under the LPAD pathway, labeling for Arikayce includes certain statements to convey that the drug has been shown to be safe and effective only for use in a limited population.
Arikayce also was approved under the accelerated approval pathway. Under this approach, FDA may approve drugs for serious or life-threatening diseases or conditions where the drug is shown to have an effect on a surrogate endpoint that is likely to predict a clinical benefit to patients. The approval of Arikayce was based on achieving three consecutive negative monthly sputum cultures by month six of treatment. FDA states it requires an additional, post-market study to be conducted by the drug’s sponsor describing the clinical benefits of Arikayce.
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."