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FDA approves Arikayce to treat lung disease
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The FDA announced it has granted Insmed, Inc. approval for Arikayce, a new drug developed for the treatment of lung disease caused by Mycobacterium avium complex.
Arikayce is the first drug to be approved under the new Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD pathway).
“As bacteria continue to grow impervious to currently available antibiotics, we need to encourage the development of drugs that can treat resistant infections,” FDA Commissioner Scott Gottlieb, MD, said in a news release. “That means utilizing novel tools intended to streamline development and encourage investment into these important endeavors."
Arikayce (amikacin liposome inhalation suspension) was approved for a limited patient population for whom conventional treatment of M. avium complex is ineffective. As a requirement of approval under the LPAD pathway, the drug label includes statements to demonstrate that it is safe and effective for use only in a limited population, according to the release.
Arikayce also was approved under the accelerated approval pathway with the condition that Insmed conduct an additional postmarket study detailing the clinical benefits of the drug.
As Infectious Disease News previously reported, the passage of the 21st Century Care Act in 2016 allows “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.” Under this act, Congress established the LPAD pathway to support drug development through streamlined clinical development programs.
Additionally, the accelerated approval pathway allows the FDA to 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 reasonably likely to predict a clinical benefit to patients.”
A randomized, controlled trial demonstrated the safety and efficacy of Arikayce taken via nebulizer.
The prescribing information for Arikayce includes a boxed warning about the increased risk for respiratory conditions that, in some cases, have led to hospitalization. These conditions include hypersensitivity pneumonitis, bronchospasm, exacerbation of underlying lung disease and hemoptysis. Other side effects of Arikayce include dysphonia, cough, ototoxicity, upper airway irritation, musculoskeletal pain, fatigue, diarrhea and nausea, according to the release.
The FDA also granted Arikayce fast track, breakthrough therapy, priority review, qualified infectious disease product and orphan drug designations.
“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,” Gottlieb said. “This pathway, advanced by Congress, aims to spur 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."
Disclosure: Gottlieb reports no relevant financial disclosures.