ARIKAYCE Suspension – Treatment for Refractory MAC Lung Disease in Adults

ARIKAYCE is an inhaled liposomal suspension containing amikacin, specifically approved for treating adults with refractory Mycobacterium avium complex (MAC) lung disease. It is intended for patients who have not responded to at least six months of combination multidrug therapy and have limited or no alternative treatment options.

• Treatment of refractory Mycobacterium avium complex (MAC) lung disease in adults who do not achieve negative sputum cultures after a minimum of six consecutive months of multidrug background regimen therapy.
• Used as part of a combination antibacterial drug regimen.
• Reserved for adults with limited or no alternative treatment options due to limited clinical safety and effectiveness data.

Limitation of Use: ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.

amikacin

ARIKAYCE delivers amikacin encapsulated in liposomes directly to the lungs via inhalation. Amikacin is an aminoglycoside antibiotic that works by inhibiting bacterial protein synthesis, thereby killing or stopping the growth of Mycobacterium avium complex bacteria causing lung infection.

Adults: The recommended dose is the once-daily oral inhalation of the entire contents of one 590 mg/8.4 mL vial of ARIKAYCE using the Lamira® Nebulizer System only.

Before use, the vial should be shaken well for 10 to 15 seconds until the suspension is uniform and at room temperature. The vial is opened by flipping the top and removing the metal ring and rubber stopper, then pouring the contents into the nebulizer medication reservoir.

Important Administration Instructions:

• Use ARIKAYCE only with the Lamira Nebulizer System as specified.
• Pre-treatment with a short-acting selective beta-2 agonist (bronchodilator) is recommended for patients with hyperreactive airway disease, chronic obstructive pulmonary disease, asthma, or bronchospasm.

Missed Dose: If a dose is missed, take the next dose the following day; do not double the dose to compensate.

ARIKAYCE is contraindicated in patients with known hypersensitivity to any aminoglycoside antibiotic.

Respiratory Adverse Reactions

• Hypersensitivity pneumonitis has been reported and may require discontinuation and corticosteroid treatment.
• Hemoptysis (coughing up blood) and bronchospasm can occur with a higher frequency; manage medically if these events occur.
• Exacerbation of underlying pulmonary disease such as COPD or bronchiectasis has been reported more often with ARIKAYCE.

Serious Hypersensitivity

• Anaphylaxis and other life-threatening hypersensitivity reactions may occur; discontinue ARIKAYCE and provide appropriate supportive care if these develop.

Ototoxicity

• Includes symptoms such as deafness, tinnitus, dizziness, vertigo, and presyncope, with a higher frequency compared to background therapy.
• Close monitoring of patients with auditory or vestibular dysfunction is important; discontinue if ototoxicity develops.
• Patients with certain mitochondrial DNA variants (e.g., MT-RNR1 m.1555A>G) are at increased risk, even at therapeutic serum levels.

Nephrotoxicity

• Observed but not more frequent than background therapy; aminoglycosides are known to cause kidney damage.
• Close monitoring is advised in patients with existing or suspected renal impairment.

Neuromuscular Blockade

• May worsen muscle weakness in patients with neuromuscular disorders like myasthenia gravis.
• If neuromuscular blockade occurs, it may be reversed with calcium salts but respiratory support may be necessary.

Embryo-Fetal Toxicity

• Use in pregnancy may cause total, irreversible, bilateral congenital deafness in the fetus.
• Women who are pregnant or may become pregnant should be informed about the risks.

Common adverse reactions (≥10% incidence in refractory MAC lung disease patients) include:

• Dysphonia (voice changes)
• Cough
• Bronchospasm
• Hemoptysis
• Musculoskeletal pain
• Upper airway irritation
• Ototoxicity-related symptoms (tinnitus, dizziness)
• Fatigue or asthenia
• Exacerbation of underlying pulmonary disease
• Diarrhea
• Nausea
• Headache

Serious adverse reactions: Hypersensitivity pneumonitis, anaphylaxis, nephrotoxicity, and neuromuscular blockade have been reported.

The label does not provide specific drug interaction information for ARIKAYCE; however, aminoglycosides may interact with neuromuscular blockers and other nephrotoxic or ototoxic drugs. Careful monitoring is advised.

Pregnancy:

Use of ARIKAYCE during pregnancy may cause fetal harm, including total, irreversible, bilateral congenital deafness. Inform women of childbearing potential about these risks.

Lactation:

The label does not provide specific information on use during breastfeeding.

Specific overdose information is not provided. In general, aminoglycoside overdose may cause ototoxicity, nephrotoxicity, and neuromuscular blockade, which require medical management.

Store ARIKAYCE at room temperature. Before use, ensure the suspension is well mixed and at room temperature for proper nebulization. Do not freeze.