FDA approved Arikayce (amikacin liposome inhalation suspension) for Refractory MAC Lung DiseaseFDA approved new drug Arikayce for hard to treat lung infection.

FDA From the archive Source published September 28, 2018 Summary added July 1, 2026 DOI ↗ Editorial oversight: Dr. Amelia Tan, PhD · Internal Medicine & Chronic Disease

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Key Takeaway

Consider Arikayce only for refractory MAC lung disease after 6 months of failed background therapy, with limited alternatives.

The FDA has approved Arikayce (amikacin liposome inhalation suspension) for a limited population of adults with refractory Mycobacterium avium complex (MAC) lung disease. The drug is indicated for patients who have not achieved negative sputum cultures after at least 6 consecutive months of a multidrug background regimen and who have limited or no alternative treatment options. Arikayce is to be used as part of a combination antibacterial drug regimen. The approval is based on accelerated approval using the surrogate endpoint of sputum culture conversion (3 consecutive negative monthly cultures) by Month 6. Clinical benefit, such as improvement in symptoms or survival, has not yet been established. Continued approval may depend on confirmatory trials. Clinicians should note that Arikayce has only been studied in refractory MAC lung disease and is not recommended for non-refractory disease.

＋ Clinical Details (Mechanism · Dosing · Trial Data · Warnings)

Mechanism of Action

Not reported in label.

Indication & Patient Population

Arikayce is indicated in adults who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Limitation of Use: Arikayce has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of Arikayce is not recommended for patients with non-refractory MAC lung disease.

Dosing & Administration

For oral inhalation use only. Use Arikayce vials only with the Lamira Nebulizer System. Pre-treatment with inhaled bronchodilator should be considered in patients with a history of hyperreactive airway disease. The recommended dosage in adults is once daily oral inhalation of the contents of one 590 mg/8.4 mL Arikayce vial. If a dose is missed, administer the next dose the next day; do not double the dose.

Key Clinical Trial Data

Trial 1 (NCT#02344004) was an open-label, randomized (2:1), multi-center trial in patients with refractory MAC lung disease. Patients were randomized to Arikayce plus a background regimen or background regimen alone. The surrogate endpoint was culture conversion (3 consecutive monthly negative sputum cultures) by Month 6. A total of 336 patients were randomized (Arikayce plus background regimen, n=224; background regimen alone, n=112). Mean age was 64.7 years. Further details not available in label.

Warnings & Contraindications

Not reported in label.

Place in Therapy

Arikayce is reserved for adults with refractory MAC lung disease who have limited or no alternative treatment options. It should be used as part of a combination antibacterial drug regimen. Clinical benefit has not been established; approval is based on a surrogate endpoint.

The FDA has approved a new drug called Arikayce (amikacin liposome inhalation suspension) for a specific group of adults with a lung infection known as Mycobacterium avium complex (MAC) lung disease. This infection can be hard to treat, and Arikayce is meant for patients who have not had success with at least 6 months of other antibiotic treatments. The drug is inhaled and is used along with other antibiotics.

Arikayce was approved using an accelerated process, which means the FDA looked at a surrogate endpoint: whether the drug could clear the bacteria from sputum samples for three months in a row. While this is a promising sign, it is not yet proven that the drug improves symptoms or helps people live longer. The manufacturer must do more studies to confirm the drug's benefits.

This approval is only for patients with refractory MAC lung disease, meaning their infection has not responded to standard treatment. It is not for people with newly diagnosed or less severe forms of the disease. If you or a loved one has MAC lung disease, talk to your doctor about whether Arikayce might be an option. Your doctor can help you understand the potential benefits and risks based on your specific situation.

What this means for you:

Arikayce is a new inhaled antibiotic for stubborn MAC lung infections, but its long term benefits are still being studied.

Study Details

Study typeFda approval

PublishedSep 2018

View Original Abstract ↓

1 INDICATIONS AND USAGE LIMITED POPULATION: ARIKAYCE ® is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options . This drug is indicated for use in a limited and specific population of patients. This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established [see Clinical Studies (14) ] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials . LIMITED POPULATION: ARIKAYCE is an aminoglycoside antibacterial indicated in adults who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients. ( 1 ) This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. ( 1 ) Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease. Limitation of Use : ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.

FDA approved Arikayce (amikacin liposome inhalation suspension) for Refractory MAC Lung DiseaseFDA approved new drug Arikayce for hard to treat lung infection.

Study Details

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FDA approved Arikayce (amikacin liposome inhalation suspension) for Refractory MAC Lung DiseaseFDA approved new drug Arikayce for hard to treat lung infection.

Study Details

Intrawound tobramycin plus vancomycin did not reduce deep infection risk compared with vancomycin alone in high-risk tibial fractures

Adding Antibiotics to Bone Surgery Didn't Lower Infection Risk

Clinical research that matters. Delivered to your inbox.

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