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The US Food and Drug Administration today approved a new drug, Arikayce (suspension of amikacin liposome inhalation), to treat a lung disease caused by a group of bacteria Mycobacterium avium complex (MAC). do not respond to conventional treatment (refractory disease).
MAC is a type of non-tuberculous mycobacteria that is commonly found in water and soil. Symptoms of the disease in MAC patients include persistent cough, fatigue, weight loss, night sweats, and sometimes shortness of breath and coughing.
"As bacteria continue to become insensitive to currently available antibiotics, we need to encourage the development of drugs that can treat resistant infections, which means using innovative tools to streamline development and encourage investment," said Scott Gottlieb, Commissioner the FDA. MD "This approval is the first time that a drug is approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs and is an important political step.This path, advanced by Congress, aims to stimulate development of drugs We see a lot of interest from sponsors in using this new pathway, and we hope that it will further stimulate the development and approval of antibacterial drugs for the treatment of serious or life-threatening infections in patients limited populations of unmet medical needs. "
Arikayce is the first drug to be approved as part of the limited pathway for antibacterial and antifungal drugs, or LPAD, established by Congress in the 21st century to promote the development and approval of antibacterial and antifungal drugs intended to treat serious or fatal infections. in a limited population of patients with unmet needs. Approval as part of 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 approved drugs under the LPAD pathway, the labeling for Arikayce includes certain statements to indicate that the drug has proven safe and effective only for a limited population.
Arikayce has also been approved as part of the accelerated approval process. As part of this approach, the FDA may approve drugs for serious or life-threatening diseases or conditions for which it has been demonstrated that the drug has an effect on a surrogate endpoint that can predict a clinical benefit to patients. Arikayce's approval was based on obtaining three consecutive negative monthly sputum cultures in the sixth month of treatment. The FDA will ask the sponsor of Arikayce to conduct an additional post-marketing study to describe the clinical benefits of Arikayce.
The safety and effectiveness of Arikayce, an inhaled, nebulized treatment, have been demonstrated in a randomized controlled clinical trial in which patients were divided into one of two treatment groups. . One group of patients received Arikayce plus a multi-drug basic antibacterial treatment, while the other treatment group received a multi-drug antibacterial treatment alone. At the sixth month of treatment, 29% of Arikayce-treated patients had no growth of mycobacteria in their sputum cultures for three consecutive months, compared to 9% of patients not treated with Arikayce.
Arikayce's prescribing information includes a boxed warning regarding the increased risk of respiratory disorders, including hypersensitivity pneumonitis (inflamed lungs), bronchospasm (tightness of the airways), exacerbation of Underlying pulmonary diseases and hemoptysis cases The other common side effects in patients taking Arikayce were dysphonia (difficulty speaking), cough, ototoxicity (impaired hearing), upper respiratory tract irritation, pain musculoskeletal, fatigue, diarrhea and nausea.
The FDA has assigned Fast Track, Breakthrough Therapy, Priority Review, and Qualified Infectious Disease Product (QIDP) to this application. The QIDP designation is given to antibacterial products that treat serious or life-threatening infections under the FDA Safety and Innovation Act's Generating Antibiotic Incentive Now (GAIN) title. Arikayce has also received the orphan drug designation, which provides additional incentives to help and encourage the development of drugs for rare diseases.
The FDA has approved Arikayce's approval at Insmed, Inc. of Bridgewater, New Jersey.
The FDA, an agency of the US Department of Health and Human Services, protects public health by ensuring the safety, efficacy and safety of drugs, vaccines and other biological products for human and human use. The agency is also responsible for the security of our country's food supply, cosmetics, food supplements, products emitting electronic radiation and the regulation of tobacco products.
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