ARIKAYCE Amikacin 590 mg/8.4mL Suspension, 28 vials — NDC 71558-590-28 (Billing 71558-0590-28)

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.

2 DOSAGE AND ADMINISTRATION For oral inhalation use only. ( 2.1 ) Use ARIKAYCE vials only with the Lamira Nebulizer System. ( 2.1 ) Pre-treatment with inhaled bronchodilator should be considered in patients with a history of hyperreactive airway disease. ( 2.1 ) The recommended dosage in adults is once daily oral inhalation of the contents of one 590 mg/8.4 mL ARIKAYCE vial. ( 2.2 )

2.1Important Administration Instructions ARIKAYCE is for oral inhalation use only. Administer by nebulization only with the Lamira ® Nebulizer System. Refer to the Instructions for Use for full administration information on use of ARIKAYCE with the Lamira Nebulizer System.

Instruct patients using a bronchodilator ('reliever') to first use the bronchodilator following the bronchodilator leaflet for use information before using ARIKAYCE. Pre-treatment with short-acting selective beta-2 agonists should be considered for patients with known hyperreactive airway disease, chronic obstructive pulmonary disease, asthma, or bronchospasm [see Warnings and Precautions (5.3) ] .

2.2Recommended Dosage The recommended dosage of ARIKAYCE in adults is once daily inhalation of the contents of one 590 mg/8.4 mL ARIKAYCE vial (590 mg of amikacin) using the Lamira Nebulizer System [see Clinical Studies (14) ] . Administer ARIKAYCE with the Lamira Nebulizer System only. ARIKAYCE should be at room temperature before use.

Prior to opening, shake the ARIKAYCE vial well for at least 10 to 15 seconds until the contents appear uniform and well mixed. The ARIKAYCE vial is opened by flipping up the plastic top of the vial then pulling downward to loosen the metal ring. The metal ring and the rubber stopper should be removed carefully.

The contents of the ARIKAYCE vial can then be poured into the medication reservoir of the nebulizer handset. If a daily dose of ARIKAYCE is missed, administer the next dose the next day. Do NOT double the dose to make up for the missed dose.

3 DOSAGE FORMS AND STRENGTHS ARIKAYCE is supplied as a sterile, white, milky, aqueous, liposome suspension for oral inhalation in a unit-dose glass vial containing amikacin 590 mg/8.4 mL (equivalent to amikacin sulfate 788 mg/8.4 mL). ARIKAYCE is supplied as a sterile, aqueous, liposome suspension for oral inhalation in a unit-dose glass vial containing amikacin 590 mg/8.4 mL. ( 3 )

17 PATIENT COUNSELING INFORMATION Advise the patient to read the FDA-approved patient labeling (Medication Guide and Patient Instructions for Use). Important Instructions for Administration of ARIKAYCE Instruct patients to read the Instructions for Use before starting ARIKAYCE. Instruct patients to only use the Lamira ® Nebulizer System to administer ARIKAYCE.

Advise the patient or caregiver not to use the Lamira Nebulizer System with any other medicine. Hypersensitivity Pneumonitis and Bronchospasm (Difficulty Breathing) Advise patients to inform their healthcare provider if they experience shortness of breath or wheezing after administration of ARIKAYCE. Advise patients with a history of reactive airway disease, asthma, or bronchospasm, to administer ARIKAYCE after using a short-acting bronchodilator [see Warnings and Precautions (5.1 , 5.3) ].

Hemoptysis or Cough Advise patients to inform their healthcare provider if they cough up blood or experience episodic cough either during or after ARIKAYCE administration particularly in the first month after starting ARIKAYCE [see Warnings and Precautions (5.2) and Adverse Reactions (6.1) ]. Exacerbations of Underlying Pulmonary Disease Advise patients to inform their healthcare provider if they experience worsening of their lung disease after starting ARIKAYCE [see Warnings and Precautions (5.4) ]. Dysphonia or Difficulty Speaking Advise patients to inform their healthcare provider if they have difficulty speaking.

Difficulty speaking or loss of ability to speak has been reported with ARIKAYCE [see Adverse Reactions (6.1) ]. Anaphylaxis and Hypersensitivity Reactions Advise patients and caregivers that serious and potentially life-threatening hypersensitivity reactions, that require immediate treatment could occur. Advise the patient to discontinue ARIKAYCE and seek immediate medical attention if any signs or symptoms of a hypersensitivity reaction occur [see Warnings and Precautions (5.5) ].

Ototoxicity (Ringing in the Ears) Advise patients to inform their healthcare provider if they experience ringing in the ears, dizziness, or any changes in hearing because ARIKAYCE has been associated with hearing loss [see Warnings and Precautions (5.6) ] . Advise the patient not to operate heavy machinery or do dangerous activities while inhaling ARIKAYCE through the Lamira Nebulizer System because ARIKAYCE can cause symptoms such as dizziness or respiratory symptoms. Nephrotoxicity or Kidney Damage Advise patients to inform their health care provider if they have kidney problems because kidney damage has been reported with aminoglycosides [see Warnings and Precautions (5.7) ] .

Neuromuscular Blockade Advise patients to inform their healthcare provider of known neuromuscular disease (e.g., myasthenia gravis) [see Warnings and Precautions (5.8) ]. Embryo-Fetal Toxicity Advise pregnant women that aminoglycosides, including ARIKAYCE, may cause irreversible congenital deafness when administered during pregnancy [see Warnings and Precautions (5.9) and Use in Special Populations (8.1) ] .

This Medication Guide has been approved by the U.S. Food and Drug Administration Issued: 12/2025 MEDICATION GUIDE ARIKAYCE (ar' i kase) LIMITED POPULATION (amikacin liposome inhalation suspension) for oral inhalation use Important: For oral inhalation only. What is the most important information I should know about ARIKAYCE?

ARIKAYCE can cause serious side effects, including: allergic inflammation of the lungs: These respiratory problems may be symptoms of allergic inflammation of the lungs and often come with: fever coughing fast breathing wheezing shortness of breath coughing up of blood (hemoptysis): Coughing up blood is a serious and common side effect of ARIKAYCE. severe breathing problems : Severe breathing problems can be symptoms of bronchospasm. Bronchospasm is a serious and common side effect of ARIKAYCE. Bronchospasm symptoms include: shortness of breath wheezing difficult or labored breathing coughing or chest tightness worsening of chronic obstructive pulmonary disease (COPD): This is a serious and common side effect of ARIKAYCE. serious allergic reactions: Serious allergic reactions that may lead to death have happened to people who take ARIKAYCE.

Stop taking ARIKAYCE right away and get emergency medical help if you have any of the following symptoms of a serious allergic reaction: hives itching redness or blushing of the skin (flushing) swollen lips, tongue or throat trouble breathing or wheezing shortness of breath noisy high-pitched breathing (stridor) cough nausea vomiting diarrhea feel cramps in your stomach area fast heart rate feeling light headed feeling faint loss of control of the bowels or bladder (incontinence) dizziness While using ARIKAYCE these side effects may become serious enough that treatment in a hospital is needed.

Call your healthcare provider or get medical help right away if you have any of these serious side effects while taking ARIKAYCE. Your healthcare provider may ask you to stop using ARIKAYCE for a short period of time or completely stop using ARIKAYCE. What is ARIKAYCE?

ARIKAYCE is a prescription medicine used to treat adults with refractory (difficult to treat) Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug treatment plan (regimen). It is not known if ARIKAYCE is safe and effective in children younger than 18 years of age. This product was approved by FDA using the Limited Population pathway.

This means FDA has approved this drug for a limited and specific patient population, and studies on the drug may have only answered focused questions about its safety and effectiveness. Do not use ARIKAYCE if you: are allergic to any aminoglycoside, or any of the ingredients in ARIKAYCE. See " What are the ingredients in ARIKAYCE? " at the end of this leaflet for a complete list of ingredients in ARIKAYCE.

Before using ARIKAYCE, tell your healthcare provider about all of your medical conditions, including if you: have asthma, chronic obstructive pulmonary disease (COPD), shortness of breath or wheezing (bronchospasm). have been told you have poor lung function. have or have had hearing problems (including noises in your ears such as ringing or hissing), hearing loss, or your mother has had hearing problems after taking an aminoglycoside. have been told you have certain gene variants (a change in the gene) related to hearing abnormalities inherited from your mother. have dizziness or sense of the room spinning. have kidney problems. have neuromuscular disease such as myasthenia gravis. are pregnant or plan to become pregnant.

It is not known if ARIKAYCE can harm your unborn baby. ARIKAYCE is in a class of medicines that may be connected with complete deafness in babies at birth. The deafness affects both ears and cannot be changed. are breastfeeding or plan to breastfeed.

It is not known if the medicine in ARIKAYCE passes into your breast milk and if it can harm your baby. Talk to your healthcare provider about the best way to feed your baby during t… [Excerpted — this section continues on DailyMed.]

Instructions for Use ARIKAYCE ® LIMITED POPULATION (amikacin liposome inhalation suspension) For oral inhalation use Lamira ® Nebulizer System Before using your Lamira Nebulizer System, be sure you read and understand the detailed information in the full Instructions for Use that comes with the Lamira Nebulizer System. This will provide more complete information about how to put together (assemble), prepare, use, clean, and disinfect your Lamira Nebulizer System. If you do not understand any part of the instructions, contact Insmed inLighten™ Patient Support at 1-833-LIGHT-00 (1-833-544-4800) before using the Lamira Nebulizer System.

Gather your ARIKAYCE medicine. The ARIKAYCE 28-day kit contains: 1 ARIKAYCE Quick Start Guide 1 Instructions for Use insert 1 Full Prescribing Information insert 1 Lamira Nebulizer Handset 4 Lamira Aerosol Heads (1 in each weekly box) 28 vials (1 vial each day) of ARIKAYCE (7 in each weekly box) Check to make sure you have all the necessary parts for your Lamira Nebulizer System: a. Carrying Case b.

Connection Cord c. Controller d. A/C Power Supply e. "AA" Batteries Spare Lamira Nebulizer Handset: f.

Medication Cap and Seal g. Medication Reservoir h. Blue Valve i.

Aerosol Chamber j. Mouthpiece k. Spare Aerosol Head You will also need the following supplies that do not come in your ARIKAYCE 28-day kit that will help you care for your Lamira Nebulizer System: Clear liquid soap for cleaning the Handset and Aerosol Head Distilled water for disinfecting the Handset and Aerosol Head Choose your power supply and get it ready. a.

4 "AA" batteries or b. A/C Power Supply Plug the A/C Power Supply into the Controller. Plug the A/C Power Supply into the wall outlet.

Do not insert the A/C Power into the front of the Controller. Cleaning and Disinfecting Before first use, rinse, clean, and disinfect your Handset and Aerosol Head. Moving forward, rinse, clean, and disinfect the Handset including the Aerosol Head after every use.

When you receive your Handset and Aerosol Head, they will not be sterile. Cleaning and disinfecting your Handset and Aerosol Head is important to reduce the risk of infection, illness, and contamination. 1.

Cleaning the Handset and Aerosol Head Reminder: Before first use, rinse and clean the Handset and Aerosol Head. Moving forward, rinse and clean the Handset including the Aerosol Head right after each use. • Take apart (disassemble) the Handset for cleaning • Gently wipe away any drops of medicine from the Medication Reservoir (a), Aerosol Chamber (b), and Mouthpiece (c) before rinsing, to reduce antibiotics added to water systems. Use only plain, dry paper towels or wipes.

Do not use towels or wipes that have any chemicals added to them such as alcohol, lotion, or baby wipes. Be careful not to harm the parts. Do not wipe Aerosol Head.

Throw away paper towels by disposing in trash with solid waste. • Rinse each of the parts under warm running tap water for 10 seconds . Rinse the Aerosol Head for 10 seconds on each side. Clean all Handset parts by adding a few drops of clear liquid dish soap and warm tap water to a clean tub or bowl.

Cover the Handset parts in the warm soapy water and soak for 5 minutes , shaking them periodically. Then rinse them thoroughly under warm running tap water. 2.

Disinfecting the Handset and Aerosol Head Before First Use Reminder: Disinfect the Handset and Aerosol Head before first use. Be sure your Handset and Aerosol Head are clean before you disinfect. Make sure the pot has enough distilled water to completely cover all the Handset parts, including the Aerosol Head.

Heat the water to a boil in a clean pot. Place the Handset parts, including the Aerosol Head, into the boiling water. Boil for a full 5 minutes .

Note: It may be helpful to use a timer. Air dry on a lint-free towel. When fully dry, wrap up the parts in a lint-free towel for storage.

You can put them together again just before taking your next treatment. Assembling Your Handset Step 1: Wash your… [Excerpted — this section continues on DailyMed.]