FDA grants Priority Review to Insmed’s ARIKAYCE sNDA for full approval in MAC lung disease, supported by positive Phase 3b ENCORE results.
Written By: Rishabh Sonawane, BPharm
Reviewed By: Pharmacally Editorial Team
Insmed’s ARIKAYCE (amikacin liposome inhalation suspension) has received FDA Priority Review for a supplemental New Drug Application (sNDA) seeking full approval for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial regimen. The FDA set a January 28, 2027, PDUFA target date, with the application supported by positive results from the Phase 3b ENCORE study.
FDA Review Could Expand ARIKAYCE Use Earlier in Disease
ARIKAYCE received accelerated FDA approval in 2018 for refractory MAC lung disease and became the first product approved through the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD).
The new sNDA seeks full approval for ARIKAYCE as part of a combination antibacterial regimen, potentially expanding its use beyond patients with refractory disease to patients earlier in the course of MAC lung infection.
Full approval would also fulfill the post-marketing requirement associated with the drug’s accelerated approval.
ARIKAYCE uses a liposomal formulation of amikacin to deliver the aminoglycoside antibiotic directly to the lungs through inhalation. The formulation is administered alongside other antibacterial agents rather than as monotherapy.
ENCORE Met Respiratory and Culture Conversion Endpoints
The Phase 3b ENCORE study (NCT04677569) enrolled 425 adults with a new occurrence of MAC lung infection who had not previously received antibiotics for the infection. The randomized, double-blind, placebo-controlled study was conducted across 177 sites globally.
Patients received either once-daily ARIKAYCE plus multidrug therapy or placebo plus multidrug therapy for 12 months, followed by three months off treatment to assess the durability of culture conversion.The treatment groups included:
- ARIKAYCE plus multidrug therapy: 213 patients
- Placebo plus multidrug therapy: 212 patients
- Treatment duration: 12 months, followed by three months off treatment
The primary endpoint was the change from baseline in Respiratory Symptom Score at Month 13. ENCORE met its primary endpoint, with ARIKAYCE plus multidrug therapy producing a statistically significant improvement in respiratory symptoms compared with multidrug therapy alone.
The study also met its multiplicity-controlled secondary culture conversion endpoints. Patients receiving ARIKAYCE achieved earlier, greater and more durable culture conversion than those receiving comparator therapy.
The safety profile remained consistent with the established profile of ARIKAYCE, with no new safety signals identified.
Respiratory Risks Remain Central to ARIKAYCE Safety
ARIKAYCE carries a boxed warning for serious respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm and exacerbation of underlying pulmonary disease. These reactions have resulted in hospitalizations in some cases.
As an aminoglycoside, amikacin is also associated with class-related risks including nephrotoxicity, ototoxicity such as hearing loss and tinnitus, and neuromuscular blockade. However, the principal boxed warning for ARIKAYCE focuses on serious respiratory adverse reactions associated with the inhaled formulation.
These safety considerations remain particularly relevant if the drug moves into an earlier treatment setting involving patients with newly occurring MAC infection.
FDA Decision Expected in January 2027
Insmed Chief Medical Officer Martina Flammer said earlier treatment could be clinically important because persistent MAC infection can progressively damage lung tissue and become more difficult to treat.
The FDA granted Priority Review because the application could provide a significant improvement in safety or effectiveness for a serious condition if approved. The agency is scheduled to make its decision by January 28, 2027, under the PDUFA timeline.
Insmed also plans to discuss the ENCORE findings with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) in the fourth quarter of 2026 to support a potential expansion of the ARIKAYCE label in Japan.
The FDA review represents the next regulatory step toward converting ARIKAYCE’s accelerated approval into full approval supported by confirmatory Phase 3b evidence.
Reference
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, Insmed, 21 September 2026
Study to Evaluate ALIS (Amikacin Liposome Inhalation Suspension) in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex (ENCORE), ClinicalTrials.gov ID NCT04677569
About the Writer
Rishabha Sonawane, B.Pharm (LinkedIn) is healthcare writer with a strong interest in medical writing, regulatory affairs, clinical research, and AI-driven drug discovery. He has completed specialized training from the NIH and ICMR in clinical pharmacology, clinical research, and scientific writing. Passionate about evidence-based healthcare communication, he focuses on translating complex scientific research into clear, accurate, and engaging medical content.