The US Food and Drug Administration has approved Arikayce
(liposomal amikacin for inhalation | ALIS) for the treatment of lung disease caused by Mycobacterium avium
complex (MAC) bacteria in a limited population of patients who do not respond to conventional treatment. It is an inhaled treatment that is taken through a nebulizer.
The approval of ALIS was based on the results of the CONVERT study, a randomized, open-label, global phase 3 trial "designed to confirm the culture conversion results seen in Insmed's phase 2 clinical trial of ALIS in patients with refractory nontuberculous mycobacterial lung disease (NTM) caused by MAC," according to a statement from Insmed
. The results indicated that when added to guideline-based therapy, once-daily ALIS improved sputum culture conversion rates by the sixth month after treatment initiation with statistical significance in patients with refractory NTM caused by MAC, compared with guideline-based therapy alone (P
<.0001). "The addition of ALIS to GBT eliminated evidence of NTM lung disease caused by MAC in sputum by month 6 in 29% of patients, compared to 9% of patients on GBT alone," according to the statement.
ALIS is the first drug to be approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs. This program was 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," according to the FDA press release on the approval. Approvals of drugs under this program may come to fruition based on the results of smaller, shorter, or fewer clinical trials. As such, labeling for this liposomal amikacin for inhalation indicates that "the drug has been shown to be safe and effective only for use in a limited population."
Furthermore, the drug was approved under the Accelerated Approval pathway, through which the FDA is able to approve drugs for some serious or life-threatening diseases or conditions, "where the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients," according to the FDA statement. Arikayce was approved based on results that indicated that it was capable of ensuring 3 consecutive negative monthly sputum cultures by the sixth month of treatment.
In the press release on the drug's approval, FDA Commissioner Scott Gottlieb, MD, stated, "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. 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 the development of drugs targeting infections that lack effective therapies. 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."
Arikayce's sponsor (Insmed, Inc) is required to conduct an additional, post-market study to describe the clinical benefits of the drug.
A Boxed Warning includes information on the increased risk of respiratory conditions including hypersensitivity pneumonitis, bronchospasm, exacerbation of underlying lung disease and hemoptysis that have led to hospitalizations in some cases, according to the FDA. Other common side effects in patients taking ALIS were dysphonia, cough, ototoxicity, upper airway irritation, musculoskeletal pain, fatigue, diarrhea and nausea.
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