Amikacin Oral Inhalation

Amikacin oral inhalation (Arikayce) is approved for a specific and limited group of patients with a difficult-to-treat bacterial lung infection.

• Arikayce is indicated for adults with refractory Mycobacterium avium complex (MAC) lung disease — meaning the infection did not respond to at least 6 consecutive months of a standard combination antibiotic regimen
• It is used as part of a multidrug combination regimen, not alone
• Arikayce is reserved for adults who have limited or no other treatment options
• Arikayce is not recommended for patients with non-refractory MAC lung disease (i.e., patients whose infection has not yet failed standard therapy)
• This approval is based on a surrogate measure (conversion to negative sputum cultures); direct clinical benefit has not yet been fully established and may depend on results of ongoing confirmatory trials

Amikacin is an aminoglycoside antibiotic. It works by latching onto a structure inside bacterial cells called the ribosome — the machinery bacteria use to make proteins. By blocking this process, amikacin kills the bacteria. When inhaled as Arikayce, the drug is delivered directly into the lungs in high concentrations, targeting the MAC infection where it lives in the airways.

Amikacin resists breakdown by certain enzymes that inactivate related antibiotics like gentamicin and tobramycin, which can give it an advantage against some resistant organisms. However, aminoglycosides as a class — including amikacin — are not effective against Salmonella or Shigella species.

Arikayce is inhaled once daily using the Lamira Nebulizer System — the only nebulizer approved for use with this medication. The vial should be at room temperature before use and shaken well for 10–15 seconds until uniform. Pour the contents into the nebulizer handset's medication reservoir as instructed.

• Use any prescribed bronchodilator (reliever inhaler) before using Arikayce
• If a dose is missed, take the next dose the next day — never double up to make up for a missed dose
• Follow your prescriber's instructions and the Lamira Nebulizer System's Instructions for Use carefully

The most common side effects seen in clinical trials of Arikayce include:

More serious reactions — including severe lung inflammation, serious allergic reactions, and significant hearing damage — require prompt medical attention. Contact your doctor right away if your breathing worsens, you cough up blood, or you notice sudden hearing or balance changes.

• Respiratory reactions (Boxed Warning): Arikayce significantly increases the risk of bronchospasm, hypersensitivity pneumonitis (lung inflammation), hemoptysis (coughing up blood), and worsening of underlying lung disease — some leading to hospitalization. Watch closely for any breathing changes.
• Hearing and balance damage (ototoxicity): Arikayce is associated with a higher rate of hearing loss, tinnitus, and dizziness than background therapy alone. Patients with certain inherited genetic variants (MT-RNR1 gene) are at especially high risk, even at normal drug levels. Hearing should be monitored throughout treatment.
• Serious allergic reactions (anaphylaxis): Life-threatening allergic reactions have been reported. Signs include hives, swollen tongue or lips, difficulty breathing, low blood pressure, or fainting — seek emergency care immediately.
• Kidney damage (nephrotoxicity): Kidney problems were observed in clinical trials. Close monitoring is needed for patients with known kidney issues or for older adults.
• Neuromuscular blockade: Arikayce may worsen muscle weakness in patients with neuromuscular conditions such as myasthenia gravis. These patients were not included in clinical trials.
• Embryo-fetal toxicity: Arikayce can cause total, irreversible, bilateral (both-ear) congenital deafness in babies exposed before birth. Avoid use in pregnancy unless no safer alternative exists.

Arikayce has not been studied in formal drug interaction trials, but important interactions are known based on the aminoglycoside drug class:

• Other nephrotoxic, neurotoxic, or ototoxic drugs: Combining Arikayce with other medications that can harm the kidneys, nerves, or hearing increases the risk of serious damage — avoid concurrent use
• Ethacrynic acid, furosemide, urea, or intravenous mannitol (certain diuretics/water pills): These can raise amikacin levels in the blood and tissue, increasing the risk of toxicity — avoid concurrent use

Not a complete list. The label covers the interactions the manufacturer studied or reported, and our own interaction database does not cover every medicine either. Bring your full medication list, including vitamins and herbal products, to your pharmacist.

• Aminoglycoside allergy: Arikayce is contraindicated if you have ever had a hypersensitivity reaction to any aminoglycoside antibiotic (e.g., gentamicin, tobramycin, neomycin)
• Pregnancy: Aminoglycosides, including Arikayce, can cause permanent, total hearing loss in babies exposed in the womb — tell your doctor if you are pregnant or trying to conceive
• Breastfeeding: It is unknown how much Arikayce passes into breast milk after inhalation — discuss risks and benefits with your doctor
• Children: Safety and effectiveness have not been established in patients under 18 years old
• Older adults (65+): No overall difference in effectiveness, but kidney function should be monitored since kidney function often declines with age
• Hearing problems or family history of aminoglycoside-related hearing loss: Certain inherited genetic variants (MT-RNR1) raise the risk of permanent hearing loss — your doctor should consider this before starting treatment
• Neuromuscular conditions (e.g., myasthenia gravis): Arikayce may worsen muscle weakness — close monitoring is needed
• Asthma, COPD, or reactive airway disease: Pre-treatment with a short-acting bronchodilator should be considered
• Kidney impairment: Arikayce has not been formally studied in patients with kidney disease, but renal function should be monitored

Specific overdose effects with Arikayce have not been identified in studies, but if you suspect you or someone else has taken too much, stop using the medication immediately and seek medical advice. Doctors may check kidney function, and in serious cases, hemodialysis (a kidney-filtering procedure) may help remove amikacin from the body. Contact a Regional Poison Control Center for guidance.

The precise relationship between Arikayce blood levels and its effects in the body — including how much drug is needed for the best outcome or the greatest risk of side effects — has not been fully established. The exposure-response relationships and time course of pharmacodynamic response for Arikayce are currently unknown.

Because Arikayce is inhaled, it delivers very high concentrations of amikacin directly into the lungs and airways — far higher than what reaches the bloodstream. Sputum (airway mucus) concentrations are highest in the first 1–4 hours after inhalation and drop to about 5% of that peak by 48–72 hours. Blood levels are much lower than those seen with intravenous amikacin at standard doses. Amikacin is not meaningfully broken down by the body; what is absorbed into the bloodstream is cleared primarily through the kidneys (glomerular filtration), while unabsorbed drug is likely cleared by normal airway processes. The apparent serum half-life after inhalation ranges from roughly 6 to 20 hours, with considerable variability between individuals.

Store ARIKAYCE vials refrigerated at 2°C to 8°C (36°F to 46°F) until expiration date on vial. Do not freeze. stored at room temperature up to 25°C (77°F) for up to 4 weeks.