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WHEN ADULTS WITH MAC LUNG DISEASE REMAINED CULTURE POSITIVE AFTER 6 MONTHS OF MULTIDRUG THERAPY,*
ARIKAYCE helped patients get converted and stay converted^1-3

Only patients on ARIKAYCE + multidrug therapy for 12 months after conversion stayed culture negative after all treatment ended.^1,3

Primary and Secondary Charts

Primary and Secondary Charts

Primary and Secondary Charts

^aPercentage comparison.
^bP<0.0001.

^aPercentage comparison.
^bP<0.0001.

^aPercentage comparison.
^bP<0.0001.

^cNot significant.
^dP<0.002 vs multidrug therapy.
^eP<0.0001.

^fPercentage of patients remaining culture negative 12 months off all treatment was an exploratory endpoint likely reflecting the natural history of the disease.³

In the CONVERT^† trial, the endpoints of the change from baseline in 6MWT distance and SGRQ did not demonstrate clinical benefit at Month 6.¹

Only patients on ARIKAYCE + multidrug therapy for 12 months after conversion stayed culture negative after all treatment ended^1,3

In the CONVERT^† trial, the endpoints of the change from baseline in 6MWT distance and SGRQ did not demonstrate clinical benefit at Month 6.¹

CONVERT study design

The efficacy and safety of ARIKAYCE + multidrug therapy vs multidrug therapy alone were evaluated in an open-label, randomized (2:1), multicenter, global, Phase 3 trial of 336 adult patients with refractory^‡ MAC lung disease.^1,2

Primary endpoint^1,2

Patients who culture converted by Month 6. Patients needed to achieve their first negative culture by Month 4 to meet the primary endpoint.^§ Culture conversion was defined as 3 consecutive monthly negative sputum cultures. The study design required 2 or 3 negative sputum samples per month for 3 consecutive months to confirm culture conversion.

Secondary endpoints/responder analysis^2,3

Patients who remained culture converted 12 months after initial conversion, and 3 and 12 months after treatment ended, along with change from baseline in 6MWT and SGRQ.

Patient selection in the CONVERT trial

Key inclusion criteria⁴

MAC lung disease documented by at least 2 positive cultures (≥1 positive culture within 6 months prior to screening and 1 positive culture at screening with cultures obtained ≥1 month apart)
Did not respond to active therapy for ≥6 months. Treatment was either ongoing or had been stopped no more than 12 months before the screening visit
Evidence of underlying lung disease, such as nodular bronchiectasis and/or fibrocavitary disease by chest radiography or CT

Key exclusion criteria⁴

Cystic fibrosis
MAC lung disease resistant to amikacin (as identified by MIC susceptibility >64 μg/mL)
Active pulmonary malignancy (primary or metastatic) or any malignancy requiring chemotherapy or radiation therapy within 1 year before screening or anticipated during study period
Acquired and primary immunodeficiency syndrome (eg, HIV-positive patients regardless of CD4 counts)

Key baseline patient characteristics in the CONVERT trial^2,5

Key baseline patient characteristics in the convert trial

Key baseline patient characteristics in the convert trial

Parameter	ARIKAYCE + multidrug therapy (n=224) n (%)	multidrug therapy alone (n=112) n (%)
Gender
Female	165 (73.7)	68 (60.7)
Male	59 (26.3)	44 (39.3)
Underlying lung disease
Bronchiectasis only	146 (65.2)	64 (57.1)
COPD‡	29 (12.9)	19 (17.0)
COPD‡ and bronchiectasis	22 (9.8)	18 (16.1)
multidrug therapy prior to enrollment
On treatment	201 (89.7)	101 (90.2)
Off treatment for at least 3 months	23 (10.3)	11 (9.8)

See real patient experiences with ARIKAYCE

Judy, 63-year-old woman (real ARIKAYCE patient)

ARIKAYCE added to multidrug therapy after culture positive for >6 months on multidrug therapy alone and worsening symptoms¹

Judy, 63-year-old woman (real ARIKAYCE patient)

ARIKAYCE added to multidrug therapy after culture positive for >6 months on multidrug therapy alone and worsening symptoms¹

Past medical history

First diagnosed with MAC lung infection in 2008 via bronchoalveolar lavage
Failed multiple MAC treatment courses over 12 years
Chronic bronchiectasis

Microbiology after multidrug therapy

Sputum AFB positive
Amikacin susceptible: MIC 16 µg/mL
Clarithromycin susceptible: MIC 2 µg/mL
Rifabutin susceptible: MIC 0.5 µg/mL

Symptoms after multidrug therapy

Worsening cough, shortness of breath, and fatigue

Individual results may vary.

ARIKAYCE added to multidrug therapy¹

ARIKAYCE (started June 2020)
Clarithromycin
Rifabutin

Assessment and management after starting ARIKAYCE

Developed ringing in ears and hearing loss
- Temporarily stopped ARIKAYCE
- Audiology exam results normal several weeks later
Restarted ARIKAYCE, which she tolerated
Negative cultures for MAC lung disease after 12 months of treatment with ARIKAYCE + multidrug therapy

MAC treatment plan

Continue MAC treatment and monitor sputum cultures and symptoms^1,6

See Judy's story

See Judy's story

+

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.

Hi, I am Judy. My husband Mike and I live in Florida and Wisconsin. We're also very fortunate through marriage to have 10 grandchildren between us. My journey to MAC diagnosis was a long journey. I had such a bad cough, that I actually cracked a rib coughing. I went to see an infectious disease specialist and pulmonary physician who recommended a variety of tests, which led to the diagnosis of MAC.

After diagnosis with MAC, I was put on a multidrug regimen and I thought that would be the answer. I found out that my multidrug treatment wasn't working when there were multiple reoccurrences. To learn that it's not working is incredibly disheartening, and frustrating, and a little scary.

I found ARIKAYCE when I was trying to find additional tools for myself on a community blog specific to lung disease. And it just felt like a possible tool, a hopeful tool. Maybe this would work for me. And I took the ARIKAYCE product sheet to an appointment with my infectious disease specialist.

I think with ARIKAYCE, in combination with the other things that a person can do, in working with their physicians, there is an opportunity to get results, positive results. The onboarding of ARIKAYCE with an Arikares Coordinator and an Arikares Trainer, was easier than I thought also, especially as it related to the financial support. The actual training of the ARIKAYCE was pretty smooth. Was actually very smooth. The side effects that I experienced was a little bit of loss of voice, being hoarse. I had a tiny bit of nasal drip. I had a little bit of ear ringing. I knew what to expect. I had a very hands-on and collaborative team, an infectious disease specialist, a pulmonary person, pulmonary technicians, to measure the progress. They listened to me, I kept good notes, and when they didn't know an answer, they went to seek an answer.

My advice for someone that has been diagnosed with MAC on the multidrug treatment that perhaps hasn't worked for them is to explore the opportunity that adding ARIKAYCE might present for them. I think the progress with ARIKAYCE is well measured and is successful for me, personally. I feel I have more control and I'm not looking back.

IMPORTANT SAFETY INFORMATION

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 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.

Please see full Prescribing Information at ARIKAYCE.com

Individual results may vary.

See Judy's story (real ARIKAYCE patient)

+

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.

Hi, I am Judy. My husband Mike and I live in Florida and Wisconsin. We're also very fortunate through marriage to have 10 grandchildren between us. My journey to MAC diagnosis was a long journey. I had such a bad cough, that I actually cracked a rib coughing. I went to see an infectious disease specialist and pulmonary physician who recommended a variety of tests, which led to the diagnosis of MAC.

After diagnosis with MAC, I was put on a multidrug regimen and I thought that would be the answer. I found out that my multidrug treatment wasn't working when there were multiple reoccurrences. To learn that it's not working is incredibly disheartening, and frustrating, and a little scary.

I found ARIKAYCE when I was trying to find additional tools for myself on a community blog specific to lung disease. And it just felt like a possible tool, a hopeful tool. Maybe this would work for me. And I took the ARIKAYCE product sheet to an appointment with my infectious disease specialist.

I think with ARIKAYCE, in combination with the other things that a person can do, in working with their physicians, there is an opportunity to get results, positive results. The onboarding of ARIKAYCE with an Arikares Coordinator and an Arikares Trainer, was easier than I thought also, especially as it related to the financial support. The actual training of the ARIKAYCE was pretty smooth. Was actually very smooth. The side effects that I experienced was a little bit of loss of voice, being hoarse. I had a tiny bit of nasal drip. I had a little bit of ear ringing. I knew what to expect. I had a very hands-on and collaborative team, an infectious disease specialist, a pulmonary person, pulmonary technicians, to measure the progress. They listened to me, I kept good notes, and when they didn't know an answer, they went to seek an answer.

My advice for someone that has been diagnosed with MAC on the multidrug treatment that perhaps hasn't worked for them is to explore the opportunity that adding ARIKAYCE might present for them. I think the progress with ARIKAYCE is well measured and is successful for me, personally. I feel I have more control and I'm not looking back.

IMPORTANT SAFETY INFORMATION

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 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.

Please see full Prescribing Information at ARIKAYCE.com

Elisse, 67-year-old woman (real ARIKAYCE patient)

ARIKAYCE added to multidrug therapy after testing positive for MAC infection recurrence¹

Elisse, 67-year-old woman (real ARIKAYCE patient)

ARIKAYCE added to multidrug therapy after testing positive for MAC infection recurrence¹

Past medical history

Diagnosed with MAC lung disease in 2017
Non-sustained culture conversion after a year of multidrug therapy
Bronchiectasis with acute exacerbation

Microbiology after multidrug therapy

MAC positive via bronchoalveolar lavage

Symptoms after multidrug therapy

Excessive yellow sputum with cough and wheezing

Individual results may vary.

ARIKAYCE added to multidrug therapy¹

ARIKAYCE (started February 2020)
Ethambutol
Rifampin
Azithromycin

Assessment after starting ARIKAYCE

Culture negative for MAC infection with the addition of ARIKAYCE

MAC treatment plan

Continue to monitor sputum cultures and symptoms^1,6

See Elisse's story

See Elisse's story

+

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.

My name is Elisse. I live in Key Largo, Florida, with my husband. We have two children. MAC lung disease impacted me before I was getting treatment in a great way. I was coughing all night. It was miserable because I was exhausted and so was everybody else. It was very disruptive to everyday life. My journey to diagnosis started a few years before I was diagnosed. I went to my family doctor for annual checkups. For several years, I complained of a cough, a persistent cough. Every year she would tell me it was allergies...never really did anything about it. When I changed doctors, my new doctor told me that she listened to my lungs, didn't like what she heard, sent me for a chest x-ray and it came back that my lungs were damaged on the bottom, probably from pneumonia that I may have had. My pulmonologist diagnosed me and told me I tested positive for MAC. I was bewildered. I had no idea what it was. I had no idea where to turn. Having a MAC diagnosis sooner I think would have definitely helped me. The disease may not have progressed to both lungs. It may not have been as severe as it was when it was diagnosed, and I would have been able to start treatment earlier.

After the discussion with my doctor about the multidrug regimen, I was ready to fight. I was on multidrug treatment for MAC disease for 12 months. After I came off multidrug treatment, for a while I was fine and then I started coughing. So I went back to my pulmonologist who again diagnosed me with the MAC infection. I was disappointed.

The doctor told me that the good news was that there was a treatment available for people such as myself. And it was an antibiotic that was inhaled and went directly to the lungs. And I would need to take it in addition to the three antibiotics I was already taking. So he gave me some hope. I began taking ARIKAYCE. And I was empowered a little bit because I was ready to fight again. I tracked my progress through the sputum tests and the CT scans. When I had my first negative sputum test, I was thrilled. I said, well, this is great. I was prepared for the possible side effects for treatment with ARIKAYCE. I had some hoarseness in my voice for a few days. I lost my voice for about five days. Everybody's side effects are different when taking ARIKAYCE. So I would suggest for people to speak to their doctor if they're having any type of side effects.

The support I received from my doctors is when I call them, if I have a side effect or something's going on that I don't understand or that's uncomfortable, I'll call them and they'll give me advice on what to do. I've gotten support through online support groups, which helped me a lot. I am enrolled in Arikares Support Program. It's very helpful and they answer questions and solve problems I may have. My Arikares Coordinator keeps in touch with me concerning my ARIKAYCE treatment. She checks on my refills, if I need anything, if I am having any problems, and she keeps a line of communication open. It's great to know that I have someone in my corner.

If someone isn't getting answers from their initial doctor, I would persist in asking the questions. I do have to advocate and ask in-depth questions, detailed questions, until I get the answers I want. For MAC patients that have not seen results on other multidrug treatments, I would suggest they ask their doctor to look into ARIKAYCE for them. Life with ARIKAYCE has made me more hopeful for the future and I'm looking forward to great results.

IMPORTANT SAFETY INFORMATION

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 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.

Please see full Prescribing Information at ARIKAYCE.com

Individual results may vary.

See Elisse's story (real ARIKAYCE patient)

+

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.

My name is Elisse. I live in Key Largo, Florida, with my husband. We have two children. MAC lung disease impacted me before I was getting treatment in a great way. I was coughing all night. It was miserable because I was exhausted and so was everybody else. It was very disruptive to everyday life. My journey to diagnosis started a few years before I was diagnosed. I went to my family doctor for annual checkups. For several years, I complained of a cough, a persistent cough. Every year she would tell me it was allergies...never really did anything about it. When I changed doctors, my new doctor told me that she listened to my lungs, didn't like what she heard, sent me for a chest x-ray and it came back that my lungs were damaged on the bottom, probably from pneumonia that I may have had. My pulmonologist diagnosed me and told me I tested positive for MAC. I was bewildered. I had no idea what it was. I had no idea where to turn. Having a MAC diagnosis sooner I think would have definitely helped me. The disease may not have progressed to both lungs. It may not have been as severe as it was when it was diagnosed, and I would have been able to start treatment earlier.

After the discussion with my doctor about the multidrug regimen, I was ready to fight. I was on multidrug treatment for MAC disease for 12 months. After I came off multidrug treatment, for a while I was fine and then I started coughing. So I went back to my pulmonologist who again diagnosed me with the MAC infection. I was disappointed.

The doctor told me that the good news was that there was a treatment available for people such as myself. And it was an antibiotic that was inhaled and went directly to the lungs. And I would need to take it in addition to the three antibiotics I was already taking. So he gave me some hope. I began taking ARIKAYCE. And I was empowered a little bit because I was ready to fight again. I tracked my progress through the sputum tests and the CT scans. When I had my first negative sputum test, I was thrilled. I said, well, this is great. I was prepared for the possible side effects for treatment with ARIKAYCE. I had some hoarseness in my voice for a few days. I lost my voice for about five days. Everybody's side effects are different when taking ARIKAYCE. So I would suggest for people to speak to their doctor if they're having any type of side effects.

The support I received from my doctors is when I call them, if I have a side effect or something's going on that I don't understand or that's uncomfortable, I'll call them and they'll give me advice on what to do. I've gotten support through online support groups, which helped me a lot. I am enrolled in Arikares Support Program. It's very helpful and they answer questions and solve problems I may have. My Arikares Coordinator keeps in touch with me concerning my ARIKAYCE treatment. She checks on my refills, if I need anything, if I am having any problems, and she keeps a line of communication open. It's great to know that I have someone in my corner.

If someone isn't getting answers from their initial doctor, I would persist in asking the questions. I do have to advocate and ask in-depth questions, detailed questions, until I get the answers I want. For MAC patients that have not seen results on other multidrug treatments, I would suggest they ask their doctor to look into ARIKAYCE for them. Life with ARIKAYCE has made me more hopeful for the future and I'm looking forward to great results.

IMPORTANT SAFETY INFORMATION

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 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.

Please see full Prescribing Information at ARIKAYCE.com

Patients have been compensated.

WHEN ADULTS WITH MAC LUNG DISEASE REMAIN CULTURE POSITIVE AFTER 6 MONTHS OF MULTIDRUG THERAPY,

NTM Treatment Guidelines strongly recommend the addition of ARIKAYCE^1,6

Start multidrug treatment

At diagnosis, initiate a 3-drug regimen, especially for MAC^¶ patients with positive AFB sputum smears and/or cavitary lung disease.⁶

Add ARIKAYCE at 6 months

Guidelines strongly recommend adding ARIKAYCE if patients remain culture positive after 6 months of multidrug therapy.⁶

Keep patients on therapy

Guidelines recommend continuing MAC^¶ treatment for 12 months after culture conversion.⁶

Learn about the safety of ARIKAYCE

Footnotes

*Multidrug therapy was composed of an antimycobacterial regimen of at least 2 antibiotics based on the 2007 ATS/IDSA Statement or respective local guidelines. These drugs may have included, but were not limited to, azithromycin, clarithromycin, clofazimine, ethambutol, ethionamide, rifabutin, and rifampicin.^2,4,5,7

^†The CONVERT trial is referred to as "Trial 1" in the full Prescribing Information.

^‡Refractory MAC lung disease is defined as MAC patients who did not convert after ≥6 months of multidrug therapy.¹

^§Culture conversion by Month 6 was a surrogate endpoint.¹ Clinical benefit has not yet been established.

^||COPD was derived from the medical history data.²

^¶In patients with macrolide-susceptible MAC pulmonary disease.⁶

^#There was up to a 10-week window between screening and baseline during which time some patients may have achieved their first negative sputum culture. At the time of enrollment, 89.9% (302/336) of patients were either on a guideline-based regimen for MAC, or off guideline-based therapy for MAC for less than 3 months.^1,4,5

6MWT=6-minute walk test; AFB=acid-fast bacilli; ATS=American Thoracic Society; CT=computed tomography; HIV=human immunodeficiency virus; IDSA=Infectious Diseases Society of America; ITT-intent to treat; MIC=minimum inhibitory concentration; NTM=nontuberculous mycobacteria; SGRQ=St George's Respiratory Questionnaire.

References:

1. ARIKAYCE [package insert]. Bridgewater, NJ: Insmed Incorporated; 2023. 2. Griffith DE, Eagle G, Thomson R, et al; CONVERT Study Group. Amikacin liposome inhalation suspension for treatment-refractory lung disease caused by Mycobacterium avium complex (CONVERT): a prospective, open-label, randomized study. Am J Respir Crit Care Med. 2018;198(12):1559-1569. 3. Griffith DE, Thomson R, Flume PA, et al. Amikacin liposome inhalation suspension for refractory Mycobacterium avium complex lung disease: sustainability and durability of culture conversion and safety of long-term exposure. Chest. 2021;160(3):831-842. 4. Griffith DE, Eagle G, Thomson R, et al; CONVERT Study Group. Amikacin liposome inhalation suspension for treatment-refractory lung disease caused by Mycobacterium avium complex. Online data supplement. Am J Respir Crit Care Med. 2018;198(12)(suppl):E1-E28. Accessed March 3, 2023. https://www.atsjournals.org/doi/suppl/10.1164/rccm.201807-1318OC/suppl_file/griffith_data_supplement.pdf. 5. Data on file. Insmed Incorporated. Bridgewater, NJ. 6. Daley CL, Iaccarino JM, Lange C, et al. Treatment of nontuberculous mycobacterial pulmonary disease: an official ATS/ERS/ESCMID/IDSA clinical practice guideline. Clin Infect Dis. 2020;71(4):e1-e36. 7. Griffith DE, Aksamit T, Brown-Elliott BA, et al; ATS Mycobacterial Diseases Subcommittee. An official ATS/IDSA statement: diagnosis, treatment, and prevention of nontuberculous mycobacterial diseases. Am J Respir Crit Care Med. 2007;175(4):367-416.

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