ARIKAYCE Full-Approval Application Receives FDA Priority Review

The FDA has accepted for review a supplemental new drug application (sNDA) from Insmed Incorporated seeking full approval of amikacin liposome inhalation suspension (ARIKAYCE, Insmed) for Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen, the company announced. The FDA granted the sNDA Priority Review and set a target action date of January 28, 2027, under the Prescription Drug User Fee Act.

ARIKAYCE received FDA accelerated approval in 2018 for refractory MAC lung disease, the first product approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, created under the 21st Century Cures Act for serious infections with unmet needs. The sNDA, if approved, would convert that accelerated approval to full approval and fulfill an associated FDA post-marketing requirement; it is not itself an approval.

The sNDA is supported by the Phase 3b ENCORE study, a randomized, double-blind, placebo-controlled trial of 425 patients with a new MAC lung infection, randomized 1:1 to ARIKAYCE plus multidrug therapy (n=213) or placebo plus multidrug therapy (n=212) for 12 months. ENCORE met its primary endpoint, change in Respiratory Symptom Score at month 13, and its multiplicity-controlled secondary culture conversion endpoints, with statistically significant improvement versus multidrug therapy alone; safety was consistent with ARIKAYCE's known profile.

The FDA grants Priority Review to applications that, if approved, would offer a significant improvement in safety or effectiveness for a serious condition. "The FDA's acceptance of this application marks a pivotal moment in our ambition to change the treatment paradigm for MAC lung disease," said Martina Flammer, MD, MBA, chief medical officer of Insmed.

ARIKAYCE carries a boxed warning for increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease, some leading to hospitalization. The FDA has not yet acted on the sNDA.

Source: Insmed Incorporated. FDA grants Priority Review to Insmed's sNDA for ARIKAYCE (amikacin liposome inhalation suspension) for treatment of MAC lung disease with PDUFA target action date set for January 28, 2027. News release. Insmed Incorporated; September 21, 2026. Accessed September 22, 2026.