Glossary

Amikacin

[am-i-key-sin]

An antibiotic used in the treatment of infections. It is the main active ingredient in ARIKAYCE

Asthma

[az-muh]

An uncontrollable, often allergic breathing disorder that causes muscles in the airways of your lungs to tighten. This results in wheezing, coughing, and tightness in the chest

Bacteria

[bak-teer-ee-uh]

Organisms that live everywhere and are made up of different species

Bronchiectasis

[brong-kee-ek-tuh-sis]

A disease in which the lungs are damaged due to inflammation of the bronchial walls, which can create excessive coughing and mucus. The thick mucus further damages the lung cells. This triggers an immune response, which releases toxic inflammatory chemicals and can lead to further complications

COPD (chronic obstructive pulmonary disease)

[kron-ik] [uhb-struhkt-iv] [puhl-muh-ner-ee] [dih-zeez]

A lung disease that leads to poor breathing. COPD is a term that is used to include chronic bronchitis, emphysema, or a combination of both

Liposome

[lip-uh-sohm]

A microscopic sphere made of lipids surrounding a watery core that can contain a drug

MAC lung disease

[mak] [luhng] [dih-zeez]

A serious infection that affects the lungs of certain people after breathing in the Mycobacterium avium complex bacteria

Macrophage

[mak-ruh-feyj]

A type of immune cell

Multidrug regimen

[muhl-tee-druhg] [rej-uh-muhn]

A group of more than one antibiotic used to treat a condition

Mycobacterium avium complex (MAC)

[mahy-koh-bak-teer-ee-uhm] [ey-vee-uhm] [kom-pleks]

The most common species of NTM

Nebulizer

[neb-yuh-lahyz-er]

A machine that changes medication from a liquid to a mist so that it can be inhaled into the lungs

Nontuberculous mycobacteria (NTM)

[nohn-too-bur-kyuh-luhs] [mahy-koh-bak-teer-ee-uh]

A type of bacteria that is common in the environment

Particle

[pahr-ti-kuhl]

A tiny or very small bit

Specialty pharmacy

[spesh-uhl-tee] [fahr-muh-see]

A pharmacy where drugs that are not available at typical pharmacies are sold

IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING AND INDICATION ARIKAYCE is associated with: risk of increased respiratory adverse reactions including allergic inflammation of lungs, coughing up blood, severe breathing problems and worsening of COPD.

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, wheezing, coughing, shortness of breath, and fast breathing
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, difficult or labored breathing, wheezing, and 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), and 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.

Do not use ARIKAYCE if you are allergic to any aminoglycoside, or any of the ingredients in ARIKAYCE.

Before using ARIKAYCE, tell your healthcare provider about all medical conditions, including if you:

have asthma, COPD, shortness of breath, or wheezing (bronchospasm)
have been told you have poor lung function
have hearing problems, such as ringing in your ears or hearing loss
have dizziness or a 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 treatment with ARIKAYCE

Tell your healthcare provider about all the medicines you take, including prescription medicines and over-the-counter medicines, vitamins, and herbal supplements.

ARIKAYCE may cause serious side effects, including:

hearing loss or ringing in the ears (ototoxicity). Ototoxicity is a serious and common side effect of ARIKAYCE. Tell your healthcare provider right away if you have hearing loss or you hear noises in your ears, such as ringing or hissing. Tell your healthcare provider if you start having problems with balance or dizziness (vertigo)
worsening kidney problems (nephrotoxicity). ARIKAYCE is in a class of medicines which may cause worsening kidney problems. Your healthcare provider may do a blood test to check how well your kidneys are working during your treatment with ARIKAYCE
worsening muscle weakness (neuromuscular blockade). ARIKAYCE is in a class of medicines which can cause muscle weakness to get worse in people who already have problems with muscle weakness (myasthenia gravis)

The most common side effects of ARIKAYCE include: changes in voice and hoarseness (dysphonia), cough during or after a dose of ARIKAYCE, especially in the first month after starting treatment, muscle pain, sore throat, tiredness (fatigue), diarrhea, nausea, headache, fever, decreased weight, vomiting, rash, increased sputum, or chest discomfort.

These are not all of the possible side effects of ARIKAYCE. Call your doctor or pharmacist for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

What is ARIKAYCE?

ARIKAYCE is used in combination with multidrug therapy for adults who still test positive for MAC lung disease after at least 6 months on multidrug treatment alone.

ARIKAYCE 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.

ARIKAYCE was studied in adult patients. It is not known if ARIKAYCE is safe and effective in children younger than 18 years of age.