More than 3x as many patients achieved culture conversion by Month 6 with the addition of ARIKAYCE
Only patients on ARIKAYCE + standard therapy for 12 months after conversion were able to stay culture negative even after treatment ended
In adult patients with refractory MAC lung disease, Culture conversion results can last 15+ months1-3
ARIKAYCE is the first and only refractory MAC treatment that helped patients get converted and stay converted
aP<0.0001 vs standard therapy.
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 + standard therapy vs standard 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 endpoint1,2
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 analysis2,3
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.
Patients were considered to have refractory MAC lung disease if they did not achieve negative sputum cultures after a minimum duration of 6 consecutive months of standard therapy that was either ongoing or had been stopped no more than 12 months before the screening visit.1
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 trial2,5
Scroll to see full chart. ➝
| Parameter | ARIKAYCE + standard therapy (n=224) n (%) |
Standard 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) |
| Standard 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) |
Summary of standard therapy in the CONVERT trial at baseline
Standard 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.2,4-6
% of patients with ≤2 or ≥3 drugs in regimen4
ARIKAYCE + standard therapy (n=223)
Standard therapy alone (n=112)
AFTER 6 MONTHS OF TREATMENT FAILURE FOR MAC LUNG DISEASE,
Choose to add ARIKAYCE for your patients1
See MAC inside
Click here to get an inside view of a MAC lung infection in augmented reality (AR).
Using your smart device, scan the QR code to get an inside view of a MAC lung infection in augmented reality (AR).
Judy, 63-year-old woman (real patient)
ARIKAYCE added to standard therapy after culture positive for >6 months on standard therapy alone and worsening symptoms1
Judy, 63-year-old woman (real patient)
ARIKAYCE added to standard therapy after culture positive for >6 months on standard 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 standard 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 standard therapy
- Worsening cough, shortness of breath, and fatigue
ARIKAYCE added to standard 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 + standard therapy
Treatment plan
- Continue MAC treatment and monitor sputum cultures and symptoms1,7
See Judy's story
Transcript
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
Transcript
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 patient)
ARIKAYCE added to standard therapy after testing positive for MAC infection recurrence1
Elisse, 67-year-old woman (real patient)
ARIKAYCE added to standard 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 standard therapy
- Bronchiectasis with acute exacerbation
Microbiology after standard therapy
- MAC positive via bronchoalveolar lavage
Symptoms after standard therapy
- Excessive yellow sputum with cough and wheezing
ARIKAYCE added to standard therapy1
- ARIKAYCE (started February 2020)
- Ethambutol
- Rifampin
- Azithromycin
Assessment after starting ARIKAYCE
- Culture negative for MAC infection with the addition of ARIKAYCE
Treatment plan
- Continue ARIKAYCE and monitor sputum cultures and symptoms1,7
See Elisse's story
Transcript
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
Transcript
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
Suzanne, 68-year-old woman (real patient)
Remained culture negative following completion of treatment with ARIKAYCE + standard therapy
Suzanne, 68-year-old woman (real patient)
Remained culture negative following completion of treatment with ARIKAYCE + standard therapy
Past medical history
- Intermittent cough and hemoptysis starting in 2004
- MAC lung disease and bronchiectasis, diagnosed 2014
- Standard therapy (rifabutin, clarithromycin, and ethambutol) started in 2014
- Difficulty tolerating multiple combinations of MAC standard therapy
- Fatigue, anorexia, rash, diarrhea, and visual impairment
- Worsening lung function with continued intermittent hemoptysis
Microbiology after standard therapy
- Positive sputum cultures prior to adding ARIKAYCE for 5 years
ARIKAYCE added to standard therapy1
- Multiple standard therapy regimens x 9 months, until discontinued
- ARIKAYCE (started with new standard therapy August 2019)
ARIKAYCE added to standard therapy1
- Multiple standard therapy regimens x 9 months, until discontinued
- ARIKAYCE (started with new standard therapy August 2019)
Assessment after starting ARIKAYCE
- First negative culture 4 months after the addition of ARIKAYCE
- Subsequent cultures were consistently negative
- Remained on ARIKAYCE + standard therapy for 13 months after initial conversion
- Has remained culture negative since stopping all therapy in February 2021*
Treatment plan
- Continued use of vest airway clearance device
- Continue to monitor for recurrence of infection2
- Continue to see pulmonologist every 6 months
*Medical history as of July 2022.
Individual results may vary.
Joan, 72-year-old woman (hypothetical patient)
ARIKAYCE added after 6 months of standard therapy without culture conversion1
Joan, 72-year-old woman (hypothetical patient)
ARIKAYCE added after 6 months of standard therapy without culture conversion1
Past medical history
- COPD x 8 years
- Bronchiectasis (fibrocavitary) x 4 years
- MAC lung disease
Clinical presentation
- Nonproductive cough x 1 year, without improvement on standard therapy
- Dyspnea
- Weight loss (10 lb over the past year)
- Fatigue x 2 years
Current medications for MAC infection
- Ethambutol
- Azithromycin
- Rifampin
Microbiology
- Induced sputum cultures and smears consistently positive for MAC
- Macrolide susceptible: MIC 2.5 µg/mL
- Amikacin (IV) susceptible
Assessment
- MAC lung disease refractory to standard therapy
Treatment plan
- Add ARIKAYCE to multidrug regimen after 6 months of treatment failure1
- Monitor response and obtain sputum cultures every 1 to 2 months7
William, 49-year-old man (hypothetical patient)
On standard therapy for MAC lung disease for 6+ months. Improved symptoms and culture positive
William, 49-year-old man (hypothetical patient)
On standard therapy for MAC lung disease for 6+ months. Improved symptoms and culture positive
Past medical history
- Severe asthma x 30 years
- Bronchiectasis x 3 years
- MAC lung disease
Clinical presentation
- Improved fatigue since starting standard therapy
- Anorexia without weight loss
- Nonproductive cough with 1 episode of hemoptysis over the past 12 months
Current medications for MAC infection
- Azithromycin
- Ethambutol
- Clofazimine
Microbiology
- Induced sputum cultures and smears positive for MAC
- Macrolide susceptible: MIC 2.5 µg/mL
- Amikacin (IV) susceptible
Assessment
- MAC lung disease refractory to standard therapy
Treatment plan
- Add ARIKAYCE to multidrug regimen after 6 months of treatment failure1
- Guidelines recommendation is based on failure to convert sputum cultures after 6 months7
Daniela, 57-year-old woman (hypothetical patient)
On ARIKAYCE + standard therapy for MAC lung disease. Culture negative, but experiencing hoarseness and cough
Daniela, 57-year-old woman (hypothetical patient)
On ARIKAYCE + standard therapy for MAC lung disease. Culture negative, but experiencing hoarseness and cough
Past medical history
- Severe rheumatoid arthritis x 12 years
- Interstitial pneumonitis 2 years ago
- MAC lung disease x 1 year
- Standard therapy, followed by addition of ARIKAYCE after remaining culture positive on 6 months of standard therapy1
Clinical presentation
- Dysphonia
- Onset of cough immediately after ARIKAYCE administration
Current medications for MAC infection
- Azithromycin
- Ethambutol
- Rifabutin
- ARIKAYCE
Microbiology
- 2 sets of induced sputum cultures and smears were negative
- Macrolide susceptible: MIC 2.5 µg/mL
- Amikacin (IV) susceptible
Microbiology
- 2 sets of induced sputum cultures and smears were negative
- Macrolide susceptible: MIC 2.5 µg/mL
- Amikacin (IV) susceptible
Assessment
- Because Daniela culture converted on ARIKAYCE but is experiencing cough and dysphonia, treatment will be adjusted to help manage her symptoms
Treatment plan1
- Bronchodilator use before ARIKAYCE treatment
Additional management strategies8§
- Lozenges/warm water gargle postdosing/soothing fluid intake
- Change ARIKAYCE treatment to evening
- Briefly interrupt ARIKAYCE to help manage some adverse events
Helen, 58-year-old woman (hypothetical patient)
Currently culture positive after 1 year of standard therapy. Appears frustrated with recurring MAC infection
Helen, 58-year-old woman (hypothetical patient)
Currently culture positive after 1 year of standard therapy. Appears frustrated with recurring MAC infection
Past medical history
- Bronchiectasis x 6 years
- 30-pack/year smoking history, currently non-smoker
- MAC lung disease diagnosed 7 years ago
- Has experienced multiple MAC recurrences
- Culture positive after 12 months of standard treatment
Clinical presentation
- Chest pain improved slightly since starting standard therapy, but she has had worsening fatigue and cough x 8 months
Current medications for MAC infection
- Airway clearance techniques
- Azithromycin
- Rifamycin
- Ethambutol
Microbiology
- Induced sputum cultures and smears positive for MAC
- Macrolide susceptible: MIC 2.5 μg/mL
- Amikacin (IV) susceptible
Assessment
- MAC lung disease refractory to standard therapy
Treatment plan
- Add ARIKAYCE to multidrug regimen1
- Counsel patient on treatment journey and what to expect with ARIKAYCE
- Let Helen know follow-up visits may be more frequent to assess treatment
Anne, 75-year-old woman (hypothetical patient)
Currently culture positive after 8 months of standard therapy. Had difficulty tolerating some medications
Anne, 75-year-old woman (hypothetical patient)
Currently culture positive after 8 months of standard therapy. Had difficulty tolerating some medications
Past medical history
- COPD x 10 years
- Asthma x 30 years
- Cardiovascular disease
- MAC lung disease
Clinical presentation
- Productive cough keeps her awake at night x 3 months
- Fatigue x 4 months
- 8-lb weight loss over 6 months
- Appears frail
Current medications for MAC infection
- Airway clearance techniques
- Ethambutol
- Rifamycin
- Azithromycin
Microbiology
- Induced sputum cultures and smears consistently positive for MAC
- Macrolide susceptible: MIC 2.5 μg/mL
- Amikacin (IV) susceptible
Assessment
- MAC lung disease refractory to standard therapy in older patient with multiple comorbidities
Treatment plan
- Add ARIKAYCE to multidrug regimen1
- Counsel patient on treatment journey and what to expect with ARIKAYCE
- Closely monitor for adverse events and response to treatment by checking cultures every 1-2 months2
Footnotes
*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
†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
§Additional management strategies are not included in the ARIKAYCE full Prescribing Information. The data are from a telephone survey of 26 patients prescribed ARIKAYCE conducted during a 2-month period at 2 academic medical centers in the United States. Writing assistance was provided to the authors through funding from Insmed Incorporated. Insmed was not involved with the conceptualization, development, conduct, or analyses of the survey.8
6MWT=6-minute walk test; ATS=American Thoracic Society; CT=computed tomography; HIV=human immunodeficiency virus; IDSA=Infectious Diseases Society of America; ITT=intent to treat; IV=intravenous; MIC=minimum inhibitory concentration; SGRQ=St George's Respiratory Questionnaire.
References:
1. ARIKAYCE [package insert]. Bridgewater, NJ: Insmed Incorporated; 2020. 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 August 12, 2022. 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. 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. 7. 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. 8. Swenson C, Lapinel NC, Ali J. Clinical management of respiratory adverse events associated with amikacin liposome inhalation suspension: results from a patient survey. Open Forum Infect Dis. 2020:7(4). doi:10.1093/ofid/ofaa079.
Primary endpoint
More than 3x as many patients achieved culture conversion by Month 6 with the addition of ARIKAYCE1,2
More than 3x as many patients achieved culture conversion by Month 6 with the addition of ARIKAYCE1,2
Patients who met the primary endpoint at Month 6 had to have a first negative culture by Month 4.1,2
Culture conversion by Month 6 was a surrogate endpoint.1 Clinical benefit has not yet been established.
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
aP<0.0001 vs standard therapy.
SECONDARY ENDPOINTS/RESPONDER ANALYSIS
Only patients on ARIKAYCE + standard therapy for 12 months after conversion were able to stay culture negative even after treatment ended1,3
Only patients on ARIKAYCE + standard therapy for 12 months after conversion were able to stay culture negative even after treatment ended1,3
The proportion of the ITT population remaining culture negative 12 months after initial conversion, 3 months after treatment ended, and 12 months after treatment ended was 18.3%, 16.1%, and 13.4% for ARIKAYCE + standard therapy (ITT, n=224) vs 2.7%, 0%, and 0% for standard therapy alone (ITT, n=112), respectively.1,3
a
Percentage of patients remaining culture negative 12 months off all treatment was an exploratory endpoint likely reflecting the natural history of the disease.3
b
Not significant.
c
P<0.002 vs standard therapy.
d
P<0.0001.
ARIKAYCE + standard therapy (n=224)
Standard therapy alone (n=112)
Responder analysis
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Responder analysis/secondary endpoints: Percentage of patients who met the primary endpoint and remained culture negative1,3
The proportion of the ITT population (224 for ARIKAYCE + standard therapy vs 112 for standard therapy alone) who remained culture negative for 12 months after initial conversion, 3 months after treatment ended, and 12 months after treatment ended was 18.3%, 16.1%, and 13.4% for ARIKAYCE + standard therapy vs 2.7%, 0% and 0% for standard therapy alone, respectively.1,3
Responder analysis/secondary endpoints: Percentage of patients who met the primary endpoint and remained culture negative1,3
The proportion of the ITT population (224 for ARIKAYCE + standard therapy vs 112 for standard therapy alone) who remained culture negative for 12 months after initial conversion, 3 months after treatment ended, and 12 months after treatment ended was 18.3%, 16.1%, and 13.4% for ARIKAYCE + standard therapy vs 2.7%, 0% and 0% for standard therapy alone, respectively.1,3
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% of patients with ≤2 or ≥3 drugs in regimen4
% of patients with ≤2 or ≥3 drugs in regimen4
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