Of patients who remained on therapy for 12 months after culture conversion, only those on ARIKAYCE + multidrug therapy stayed culture negative after all treatment ended.1,3
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
Patients who culture converted by Month 6. Patients needed to achieve their first negative culture by Month 4 to meet the primary endpoint.ll 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.
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.
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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:
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:
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:
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
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:
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:
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:
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.
Real patients have been compensated for their time.
**Real patient data current as of October 2023.
††Real patient data current as of August 2022.
‡‡Real patient data current as of July 2024.
Guidelines strongly recommend adding ARIKAYCE if patients remain culture positive after ≥6 months of multidrug therapy.
Guidelines recommend continuing MAC treatment for 12 months after culture conversion.#
*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
†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
‡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
6MWT=6-minute walk test; AFB=acid-fast bacilli; ATS=American Thoracic Society; COPD=chronic obstructive pulmonary disease; CT=computed tomography; HIV=human immunodeficiency virus; ID=infectious disease; IDSA=Infectious Diseases Society of America; ITT=intent to treat; MIC=minimum inhibitory concentration; NTM=nontuberculous mycobacteria; SGRQ=St George's Respiratory Questionnaire.
