ARIKAYCE Liposomal: Uses, Dosage & Side Effects

What Is ARIKAYCE Liposomal and What Is It Used For?

ARIKAYCE liposomal contains the active substance amikacin, an aminoglycoside antibiotic that has been used in medicine for decades to treat serious bacterial infections. What makes ARIKAYCE unique is its innovative liposomal formulation: the amikacin is encapsulated within tiny lipid spheres called liposomes, composed primarily of dipalmitoylphosphatidylcholine (DPPC) and cholesterol. These liposomes are designed to deliver the antibiotic directly to the site of infection in the lungs, achieving high local drug concentrations while minimizing the systemic exposure that is traditionally associated with the serious side effects of aminoglycoside antibiotics.

Amikacin belongs to the aminoglycoside family of antibiotics, which includes drugs such as gentamicin, tobramycin, and streptomycin. These antibiotics work by binding irreversibly to the 30S ribosomal subunit of susceptible bacteria, interfering with messenger RNA (mRNA) reading and causing the production of abnormal, nonfunctional proteins. This disruption of protein synthesis ultimately leads to bacterial cell death. Aminoglycosides are bactericidal, meaning they kill bacteria directly rather than merely inhibiting their growth.

The liposomal delivery system of ARIKAYCE offers several key pharmacological advantages over conventional injectable amikacin. When the nebulized liposomal suspension is inhaled, the liposomes deposit on the airway epithelium and are actively taken up by alveolar macrophages — the very immune cells within which MAC bacteria preferentially reside and multiply. This targeted intracellular delivery allows the drug to reach the bacteria where they hide, potentially improving bactericidal efficacy. Furthermore, the liposomal encapsulation provides sustained release of amikacin over approximately 24 hours, supporting once-daily dosing and maintaining therapeutic concentrations at the infection site.

ARIKAYCE liposomal is specifically indicated for the treatment of nontuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium complex (MAC) in adult patients who have limited treatment alternatives and who do not have cystic fibrosis. MAC is the most common cause of NTM pulmonary disease worldwide and encompasses primarily two species: Mycobacterium avium and Mycobacterium intracellulare. MAC lung disease typically develops in individuals with underlying structural lung conditions such as bronchiectasis, chronic obstructive pulmonary disease (COPD), or prior tuberculosis, though it can also affect immunocompromised patients and, increasingly, otherwise healthy individuals, particularly postmenopausal women.

MAC lung disease is notoriously difficult to treat. Standard guideline-based therapy typically involves a combination of at least three oral antibiotics — usually a macrolide (azithromycin or clarithromycin), ethambutol, and a rifamycin (rifampicin or rifabutin) — administered for at least 12 months after achieving culture conversion (negative sputum cultures). However, treatment failure and relapse rates remain high, and many patients either do not respond to standard therapy or cannot tolerate it. ARIKAYCE liposomal was developed to address this unmet medical need, providing an additional treatment option for patients who have limited alternatives.

What Should You Know Before Using ARIKAYCE Liposomal?

Contraindications

There are specific clinical situations in which ARIKAYCE liposomal must not be used. Your doctor will have considered these before prescribing, but it is important that you understand them as well.

• Hypersensitivity: Do not use ARIKAYCE liposomal if you are allergic to amikacin, any other aminoglycoside antibiotic, soy, or any of the other ingredients in the product (cholesterol, DPPC, sodium chloride, sodium hydroxide, water for injections). Soy is included because the liposomal formulation contains lipid components derived from soy. Allergic reactions can be severe and life-threatening.
• Concurrent aminoglycoside use: Do not use ARIKAYCE liposomal if you are currently taking any other aminoglycoside antibiotic by mouth or by injection (such as gentamicin, tobramycin, or injectable amikacin). The combined effect would significantly increase the risk of ototoxicity (hearing damage), nephrotoxicity (kidney damage), and neuromuscular blockade.
• Severe renal impairment: Do not use ARIKAYCE liposomal if you have severely impaired kidney function. Although systemic absorption from inhaled liposomal amikacin is lower than from injectable amikacin, there is still some systemic exposure. Severely impaired kidneys cannot adequately eliminate amikacin from the body, leading to accumulation and an increased risk of toxic effects.

Warnings and Precautions

Before starting and during treatment with ARIKAYCE liposomal, discuss the following with your doctor:

• Bronchodilator use: If you use a bronchodilator (airway-opening medication) for breathing problems, you should use it before inhaling ARIKAYCE liposomal. This helps open the airways and allows the medication to reach deeper into the lungs.
• Kidney problems: If you have any degree of kidney impairment, your doctor may need to perform kidney function tests before starting treatment and monitor renal function regularly throughout therapy. Amikacin is primarily eliminated by the kidneys, and even with the reduced systemic exposure from inhaled liposomal delivery, kidney problems can impair clearance of the drug.
• Hearing problems or tinnitus: If you have pre-existing hearing loss, ringing in the ears (tinnitus), or balance problems (vestibular dysfunction including vertigo, dizziness, or unsteadiness), inform your doctor. You may need audiometric (hearing) testing before and during treatment. Aminoglycosides can cause irreversible damage to the cochlear hair cells, leading to permanent hearing loss, particularly at high frequencies.
• Other lung diseases: If you have other lung conditions in addition to MAC infection (such as asthma, COPD, or interstitial lung disease), tell your doctor. ARIKAYCE liposomal can cause bronchospasm and respiratory deterioration that may be more problematic in patients with pre-existing pulmonary disease.
• Neuromuscular disorders: If you have a condition that causes muscle weakness and fatigue, such as myasthenia gravis, use ARIKAYCE liposomal with caution. Aminoglycosides can worsen neuromuscular blockade and should be used with extreme care in these patients.
• Mitochondrial mutations: If you or your maternal relatives have a mitochondrial mutation disorder (a genetic condition) or have experienced hearing loss from antibiotics, inform your doctor before starting treatment. Certain mitochondrial DNA mutations, particularly the m.1555A>G mutation, dramatically increase susceptibility to aminoglycoside-induced ototoxicity. Your doctor may recommend genetic testing before initiating ARIKAYCE liposomal.

Contact your doctor immediately if, during treatment with ARIKAYCE liposomal, you experience any of the following:

• Loss of consciousness, skin rash, fever, or new or worsening breathing problems
• Deterioration of kidney function (changes in urination, swelling)
• Ear problems such as ringing in the ears, hearing loss, or balance disturbance

Pregnancy and Breastfeeding

ARIKAYCE liposomal should be avoided during pregnancy. Aminoglycosides, including amikacin, are known to cross the placenta and have been associated with irreversible bilateral congenital deafness (ototoxicity) in the developing fetus when given systemically. While the inhaled liposomal formulation results in significantly lower systemic exposure compared with injectable amikacin, the potential risk to the fetus cannot be excluded. If you are pregnant, think you may be pregnant, or are planning to become pregnant, consult your doctor before using this medication.

If you become pregnant while using ARIKAYCE liposomal, inform your doctor immediately. Your doctor will discuss with you whether to discontinue treatment, weighing the potential risk to the unborn child against the clinical benefit of continuing therapy for the MAC infection.

It is not known whether amikacin is excreted in human breast milk following inhaled liposomal administration. Given the potential for aminoglycoside-related adverse effects in the nursing infant, including ototoxicity and nephrotoxicity, your doctor will advise you on whether to discontinue breastfeeding or discontinue treatment with ARIKAYCE liposomal, taking into account the importance of the medication for your health.

Children and Adolescents

ARIKAYCE liposomal should not be used in children or adolescents under 18 years of age. The safety and efficacy of this medication have not been established in pediatric populations. MAC lung disease in children is rare and presents differently from adult disease, and the pharmacokinetics and safety profile of inhaled liposomal amikacin have not been studied in this age group.

Driving and Operating Machinery

ARIKAYCE liposomal may cause dizziness, vertigo, or other vestibular disturbances (balance problems). If you experience any of these symptoms, do not drive or operate machinery until the effects have resolved. The vestibular effects of aminoglycosides can impair coordination and spatial orientation, making activities that require alertness and balance potentially hazardous.

How Does ARIKAYCE Liposomal Interact with Other Drugs?

Drug interactions with ARIKAYCE liposomal are primarily related to the pharmacological properties of amikacin as an aminoglycoside antibiotic. Although the inhaled liposomal formulation results in lower systemic drug levels compared with parenteral amikacin, some amikacin does enter the systemic circulation, and the potential for interactions cannot be completely excluded. It is essential to tell your doctor about all medications you are currently taking, have recently taken, or plan to take, including prescription drugs, over-the-counter medicines, vitamins, and herbal supplements.

Major Interactions

• Other aminoglycosides: Concurrent use is absolutely contraindicated. Using ARIKAYCE liposomal with another aminoglycoside (e.g., gentamicin, tobramycin, streptomycin, or injectable amikacin) dramatically increases the cumulative risk of irreversible ototoxicity, nephrotoxicity, and neuromuscular blockade. An adequate washout period must be observed when switching between aminoglycoside therapies.
• Ototoxic drugs: Medications known to cause hearing damage should be used with caution alongside ARIKAYCE liposomal. These include certain loop diuretics (ethacrynic acid, furosemide), platinum-based chemotherapy agents (cisplatin, carboplatin), and other ototoxic antibiotics. The combination may increase the risk of permanent hearing loss.
• Nephrotoxic drugs: Concurrent use of medications that can damage the kidneys requires careful monitoring of renal function. Examples include non-steroidal anti-inflammatory drugs (NSAIDs), vancomycin, amphotericin B, ciclosporin, and tacrolimus. Kidney damage from these combinations may be additive or synergistic.

Minor Interactions

• Diuretics: Loop diuretics such as ethacrynic acid, furosemide, and mannitol may enhance the ototoxic and nephrotoxic effects of aminoglycosides by altering drug concentrations in the inner ear and kidneys. If you must take a diuretic, your doctor will monitor you closely.
• Neuromuscular blocking agents: Aminoglycosides can potentiate the neuromuscular blocking effects of drugs such as suxamethonium, tubocurarine, and pancuronium, potentially leading to prolonged respiratory depression or apnea. This is particularly relevant in surgical settings.
• Other drugs that reduce muscle strength: Botulinum toxin and similar agents used to treat muscle spasticity should be used with caution alongside ARIKAYCE liposomal, as the combined effect on neuromuscular function may be enhanced.

What Is the Correct Dosage of ARIKAYCE Liposomal?

ARIKAYCE liposomal should always be used exactly as prescribed by your doctor. The dosing regimen is straightforward because the medication is supplied in single-use vials, each containing a fixed dose. However, the overall treatment plan — including the accompanying multi-drug antibiotic regimen and the duration of therapy — is determined individually by your specialist physician based on your clinical response, sputum culture results, and tolerability.

Adults

Children

Elderly

How to Use ARIKAYCE Liposomal

Proper administration technique is essential for ARIKAYCE liposomal to work effectively. The medication must be inhaled using the Lamira Nebulizer System exclusively — do not use any other nebulizer device. The following steps outline the general procedure, but always refer to the detailed Instructions for Use provided with the medication and the Lamira nebulizer.

1. Preparation: Remove the vial from the refrigerator at least 45 minutes before use to allow it to reach room temperature. Do not use any other medications in the Lamira nebulizer handset.
2. Bronchodilator first: If you use a bronchodilator, inhale it before using ARIKAYCE liposomal to open your airways.
3. Shake and inspect: Shake the ARIKAYCE vial vigorously until the medication looks uniform and well mixed. The liquid should be milky white. Do not use if you notice color changes or floating particles.
4. Open the vial: Remove the orange cap, carefully pull down the metal ring until one side detaches, then remove the metal band in a circular motion. Carefully remove the rubber stopper.
5. Load the nebulizer: Pour the entire contents of the vial into the medication reservoir of the Lamira nebulizer handset and close the reservoir.
6. Inhale: Sit in a relaxed, upright position. Place the mouthpiece in your mouth and take a slow, deep breath, then breathe normally through the mouthpiece. Hold the handset level throughout treatment, which should take approximately 14 minutes (up to 20 minutes).

Each vial is for single use only. Discard any unused medication and do not save it for later use. The Lamira nebulizer handset should be used for one 28-day treatment course, and the aerosol head should be replaced every week (each 28-day package includes 4 aerosol heads).

Missed Dose

Overdose

What Are the Side Effects of ARIKAYCE Liposomal?

Like all medicines, ARIKAYCE liposomal can cause side effects, although not everyone experiences them. Many of the most frequently reported side effects are related to the inhalation route of administration and the local irritation caused by the nebulized medication in the airways. The aminoglycoside-class effects of ototoxicity and nephrotoxicity can also occur, even with the reduced systemic exposure provided by the liposomal formulation.

Understanding the Respiratory Side Effects

The respiratory side effects of ARIKAYCE liposomal are among the most commonly reported and are largely a consequence of the inhalation route of administration. Dysphonia (voice changes or difficulty speaking) is very common and may result from local irritation of the vocal cords and upper airway mucosa by the nebulized medication. Most patients find these voice changes manageable and temporary, resolving between doses or after treatment ends.

Cough and dyspnea (shortness of breath) are also very common, reflecting airway irritation and, in some cases, bronchospasm triggered by the inhaled particles. This is why bronchodilator use before ARIKAYCE inhalation is recommended for patients who already use bronchodilators. In some cases, however, respiratory deterioration can indicate a more serious inflammatory reaction in the lungs (hypersensitivity pneumonitis or eosinophilic pneumonia), which requires immediate medical evaluation and may necessitate discontinuation of treatment.

Ototoxicity and Nephrotoxicity Monitoring

Although the liposomal inhalation formulation reduces systemic exposure to amikacin compared with intravenous or intramuscular administration, clinically significant blood levels of amikacin can still be achieved, particularly with prolonged use. Therefore, regular monitoring of renal function (through blood tests measuring creatinine and estimated glomerular filtration rate) and auditory function (through audiometric testing) is essential throughout the treatment course. Patients should be instructed to report any changes in hearing (particularly high-frequency hearing loss), tinnitus, vestibular symptoms (dizziness, vertigo, unsteadiness), or changes in urination immediately.

Reporting Side Effects

If you experience any side effects while taking ARIKAYCE liposomal, talk to your doctor or pharmacist. This includes any possible side effects not listed above. You can also report side effects directly to your national medicines regulatory authority. By reporting side effects, you help provide more information on the safety of this medicine and contribute to the ongoing assessment of its benefit-risk profile.

How Should You Store ARIKAYCE Liposomal?

Proper storage of ARIKAYCE liposomal is important to maintain the integrity and efficacy of the liposomal formulation. The liposomes that encapsulate the amikacin are sensitive to temperature extremes, and improper storage can compromise the drug’s stability and performance.

• Refrigerated storage (recommended): Store the vials in a refrigerator at 2–8°C (36–46°F). This is the preferred storage condition and maintains the product’s stability until the expiration date printed on the carton and vial label.
• Room temperature storage (alternative): ARIKAYCE liposomal may alternatively be stored at room temperature up to 25°C (77°F), but only for a maximum of 4 weeks. Any unused vials must be discarded after 4 weeks at room temperature, regardless of the printed expiration date.
• Do not freeze: Freezing can damage the liposomal structure and alter the drug’s release properties. If a vial has been frozen, it must be discarded and not used.
• Before use: Remove the vial from the refrigerator at least 45 minutes before use to allow it to reach room temperature. Using cold medication directly from the refrigerator may increase airway irritation and cough.
• Keep out of reach: Store this medication out of the sight and reach of children.
• Disposal: Do not throw away medicines via wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer need. These measures help to protect the environment.

Do not use ARIKAYCE liposomal after the expiration date stated on the carton and vial label (after “EXP”). The expiration date refers to the last day of that month.

What Does ARIKAYCE Liposomal Contain?

Active Ingredient

The active substance is amikacin. Each vial contains amikacin sulfate equivalent to 590 mg amikacin in a liposomal preparation. The average dose delivered to the patient per vial during inhalation is approximately 312 mg amikacin. The difference between the vial content and the delivered dose is due to the nebulization process — some drug remains in the nebulizer device and is lost in the exhaled aerosol.

Other Ingredients (Excipients)

The other ingredients in ARIKAYCE liposomal are:

• Cholesterol: A natural lipid component of the liposomal membrane that contributes to liposome stability and controls the rate of drug release.
• Dipalmitoylphosphatidylcholine (DPPC): The primary phospholipid forming the liposomal bilayer. DPPC is a naturally occurring lung surfactant component, which contributes to the biocompatibility of the formulation with lung tissue.
• Sodium chloride: Used to adjust the tonicity (salt concentration) of the formulation to be compatible with lung tissue.
• Sodium hydroxide: Used to adjust the pH of the formulation.
• Water for injections: The aqueous vehicle for the suspension.

Appearance and Packaging

ARIKAYCE liposomal is a white to off-white, milky dispersion supplied in glass vials sealed with a rubber stopper and a metal seal with a plastic flip-lock. The medication is packaged for 28-day treatment courses: each outer carton contains 28 vials (one per day) arranged in 4 inner cartons of 7 vials each, plus 4 aerosol heads (one per week) and 1 Lamira nebulizer handset. The appearance should be inspected before each use — do not use the medication if you notice color changes or if small clumps are visible floating in the liquid after vigorous shaking.