Therapy Areas: Infectious Diseases
The US FDA approves the new antibacterial Arikayce drug to treat serious lung disease caused by MAC using a novel pathway
1 October 2018 -

US public health agency the US Food and Drug Administration on Friday announced approval for the new Arikayce drug to treat lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC) under the Limited Population Pathway for Antibacterial and Antifungal Drugs, or LPAD pathway, the accelerated approval pathway, fast track, breakthrough therapy, priority review an Qualified Infectious Disease Product (QIDP) designations.

The US FDA has granted approval of Arikayce to Insmed Inc of Bridgewater, NJ.

MAC is a type of nontuberculous mycobacteria (NTM) commonly found in water and soil. The symptoms of MAC include persistent cough, fatigue, weight loss, night sweats and occasionally shortness of breath and coughing up of blood. Arikayce will be used to treat patients with the disease who do not respond to conventional treatment.

Arikayce (amikacin liposome inhalation suspension) 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. The Approval under the LPAD pathway may be supported by a streamlined clinical development program. These programs may involve smaller, shorter or fewer clinical trials. 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.

The safety and efficacy of Arikayce, an inhaled treatment taken through a nebulizer, was demonstrated in the company's randomised, controlled clinical trial where patients were assigned to one of two treatment groups. One group of patients received Arikayce plus a background multi-drug antibacterial regimen, while the other treatment group received a background multi-drug antibacterial regimen alone.

According to the US FDA, about 29% of patients treated with Arikayce had no growth of mycobacteria in their sputum cultures for three consecutive months by the sixth month of treatment,compared to 9% of patients who were not treated with Arikayce.



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