You don’t need to go through your MAC treatment journey alone. Watch others share their experiences

Real patients and physician experts discuss their experiences with MAC lung disease and ARIKAYCE

  • ARIKAYCE Explored

    ARIKAYCE Explored

  • Judy

    Judy’s Story

  • Elisse

    Elisse’s Story

  • Lynn

    Lynn and Dr. Lapinel

  • Join a real patient on a journey to discover more about ARIKAYCE

    Travel with Judy, a real ARIKAYCE patient, as she interviews experts and patients to discuss the treatment that helped her finally test MAC-negative.

    Transcript

    I’m on this quest for knowledge to get answers for both you and me. Join me as I learn more about MAC lung disease and ARIKAYCE, from a researcher, a doctor, and a patient living with this disease. Let's get moving.

    Our first stop is at the Insmed Laboratories to learn about the science behind ARIKAYCE.

    Now the goal of Insmed, really, is to develop new therapies to treat patients with rare diseases.

    Like me.

    Yes exactly.

    Cool.

    And ARIKAYCE represents the first product that came out of these laboratories that we're going to see.

    Perfect.

    OK great.

    And here we go.

    Yep so we're entering one of our laboratories.

    So cool.

    So why don’t you grab a seat.

    Alright thank you.

    Sure.

    So David who's ARIKAYCE for?

    People with MAC infections. Before ARIKAYCE once they've exhausted other treatment options if they're still culture positive there's nothing else for them.

    Sure.

    Now, with ARIKAYCE, it gives them more hope because they can try ARIKAYCE and [it] gives them an opportunity to become MAC-negative.

    MAC-negative, that's the goal.

    That's right.

    I remember when ARIKAYCE was first FDA approved in 2018, that's a big deal, right?

    It is, it was a huge moment for us and I was here, I remember that day very well. People were jumping up and down, there was so much excitement and it really is a reflection of the service of the people here, the hard work thinking about the patients, and and it's very rewarding to know that it's finally going to help patients.

    What makes ARIKAYCE different?

    Well what makes ARIKAYCE different is it's delivered directly to the lung, the site of infection. Our formulation of ARIKAYCE uses liposomes. It's like a tiny ball.

    OK…

    You can see here, this is kind of like what it looks like.

    Right.

    But it's very very tiny, and inside the liposome is where we package all of the drug - you can see here.

    May I?

    Please. So the purple represents the drug molecules, the amikacin.

    How does it get delivered?

    So this is really very different from what you're used to, taking an oral tablet, for example.

    Right, OK.

    And how it gets delivered, is using a nebulizer and I'll show you that system.

    OK cool.

    So we have here…

    OK.

    The nebulizer, you’ve used it many times, and this was specially designed for ARIKAYCE.

    So it's a combo approach, the liposomes and the nebulizer are what helps ARIKAYCE get to the lungs.

    Exactly, so you see here, this aerosol mesh…

    OK.

    You can't see it, but there's thousands of holes inside here. They're very very tiny, and that's what creates the aerosol.

    So why mesh?

    So what the mesh does, is it pumps the liquid through these holes so you're creating droplets simultaneously and that creates a very high-density aerosol, so this is a very unique design specifically for ARIKAYCE.

    May I?

    You may, this is called the Lamira nebulizer system.

    Cool. Pretty cool actually taking it all apart and studying it and reading up on it. It wasn't as complicated as I might have thought it was initially, but I'm still impressed with it.

    You took it apart and put it together very quickly.

    I probably put it --

    Muscle memory.

    Yes. For me, I wanted to like it. I wanted to want to do it, so I bought myself a nice tray so that I had the right tools and I had tools that were attractive to me, I wanted it, I wanted it to look nice. Who wants to be reminded they're sick? I didn't want that, and I know when I have to look at it that way. I wanted it to be attractive and I wanted to want to use it. It just made it easier for me mentally to listen to my inner self and know that I could do it. And I – I’m getting choked up a little bit… to know, that your researchers and your scientists, put so much into this. So thank you. Sorry, I'm sorry. Wanna give me a hug?

    The mission of Insmed can be summed up as, simply for the patients.

    Walking around here, you can't help but see that mission come to life. It's touching and impressive to learn so many people have dedicated their time to developing ARIKAYCE, for those who had nothing else after their initial treatment.

    Let's see what we can learn at our next stop.

    Today, we're meeting with the pulmonologist in New York who's been treating MAC lung disease for over 9 years. Although she was never my doctor, she's someone I've had the pleasure of meeting a few times before this.

    One of the ways that I think I help my patients the most is by telling them that I really like to take a, a team approach to their management of care. My entire team is available, as well as myself, to be able to assist in any questions that arise, any complications that potentially may arise, so that we can make our patients feel that even though, this might be a long and complicated journey, that we have people that understand, that are informed about what they are going through, and are here to help every step of the way.

    I think the whole diagnosis of MAC lung disease and NTM can be a very scary journey especially when you're first embarking on this and I, I sure wish I had known more at the beginning. That would have helped me along the process a little more quickly. I'm not even sure I could pronounce the name of it fluently the first few months. I would imagine, Dr. Lapinel, that it's sometimes challenging to measure how much a patient is really ready to hear. So, in your particular practice, how much how do you gauge that and how do you inform your patients so that they feel cared for and supported in this journey?

    That's a wonderful question, right? Because everybody does come to the table having different backgrounds of information, everybody has different journeys, has different awareness of their underlying disease, and you want to inform people, but you also don't want to overwhelm them, um, for concern of, they may take a step back, but overall, um, you know I like to give a basic understanding of what the disease is, um, what the potential course may be, and that while we have different treatment approaches, it's tailored to that individual patient.

    I sometimes hear from other people that they don't want to start MAC treatment right away, what do you think?

    I really feel that the decision to start treatment is one that needs to be made individually. In many patients as we've learned more about the disease, I caution them that if they are not started on treatment, that it can lead to a worsening of their underlying lung disease over time. And so to that point, I follow the NTM treatment guidelines that recommend treatment over watchful waiting. And the goal of that treatment is to test MAC-negative. The guidelines do in fact discuss the length of treatment and they usually mention that patients should remain on treatment for at least 12 months from the time that they first convert to culture negative. So, somebody that has, had been MAC-positive converts to MAC-negative they must continue treatment for at least 12 months from that point. We want to do everything that we can, to help treat it, and clear it, and prevent it from coming back.

    When you say MAC lung disease is difficult to treat, like it was for me, you probably have other patients that it's the same?

    And that's why for my patients, that I do decide to start on treatment when we discuss the oral antibiotic regimen that we're putting them on, I actually bring up ARIKAYCE early on in their diagnosis. We are going to follow the guidelines and treat with proven therapies, so the addition of ARIKAYCE is strongly recommended for patients that continue to test positive after six months of treatment. These are points that are really important to know and so I really encourage patients to speak with their physicians when they're embarking on their treatment journey.

    So when you found out that ARIKAYCE was approved what was your reaction?  

    I was very happy, there was almost a sense of relief. Now we have this drug that's FDA approved, in a limited population, for patients that continue to test positive after six months of a multidrug treatment regimen.

    I think a lot of patients want to know what are the chances that ARIKAYCE is going to work for me?

    When patients ask me that, I like to refer them to the data from the study on which, for which ARIKAYCE gained you know its FDA approval. So let's head inside to my office so we can review some of the clinical data so I can answer your question a little bit more clearly.

    Thank you I'd like that.

    It was so interesting to meet Dr. Lapinel and to hear about her range of experience.

    Many patients just wanted to know what we could experience on this journey. It was refreshing to hear doctors also find this disease frustrating. After all, we're in this together.

    Let's see what we can learn at our next stop.

    We're here with Dr. Lapinel, pulmonologist, in New York. Today, she's taking us through the ARIKAYCE study and discussing the potential side effects. The place for that conversation is here in her office.

    Dr. Lapinel, I know a lot of patients want to know about ARIKAYCE and how it might affect them specifically. Can you share some of that with us?

    Yes, so I think that's a very important question. You know everybody comes to the table and wants to know how is this going to work for me? And I think to best illustrate that point it's important to look at patients who had actually participated in the ARIKAYCE study and see what their experience was. ARIKAYCE was studied in patients who had already been on a multidrug regimen and still had maintained culture positive for MAC after six months of treatment, so they look to see if ARIKAYCE was added to guideline-based therapy, if more people would test negative for MAC, versus people who were just maintained on guideline-based therapy. They also studied how long people maintained MAC-negative cultures after completing treatment. They also included some additional objectives in this study so they looked at improvement in the distance that people walked over 6 minutes, they also looked for improvement in overall health, daily life, and perceived well-being in people with lung disease as measured by a questionnaire. Overall the study showed that adding ARIKAYCE helped more people test MAC-negative and remain MAC-negative even after completing treatment, compared to people who did not add ARIKAYCE to their treatment.

    And the charts get into the details of how many people tested MAC-negative at each time point, right?

    Yes, that's correct. So if you look at the purple bar at six months, and the gray bar at six months, you'll see that 29% of people that were on ARIKAYCE with guideline-based therapy tested MAC-negative compared to 8.9% that were not on ARIKAYCE.

    So 29% compared to less than 9% after six months, is that right?

    Yes, so that's three times as many people that tested MAC-negative after adding ARIKAYCE.

    Wow, that's pretty significant.

    So Judy if you look here, at 12 months, 18.3% versus 2.7% of patients tested MAC-negative after adding ARIKAYCE, that’s seven times as many patients.

    When I first heard about ARIKAYCE and I saw this study it was really encouraging.

    We didn't have an approved treatment before ARIKAYCE for this patient population. So this data was really great to see and it meant that we had a potentially great tool to combat the disease. So, Judy, one other thing that I like to point out, is that if you look at the end of the study which was three months after completing treatment, 16.1% of patients that were on ARIKAYCE versus 0% of patients that were on [a] multidrug regimen, remained MAC-negative.

    Wow, but I'm sure some people test negative without ARIKAYCE?

    Yes, it's important to remember for many patients that are started on initial treatment, actually 2/3 of people will successfully complete treatment. ARIKAYCE was studied in the 1/3 of patients who don't get results with that first treatment.

    Dr. Lapinel, what are the chances someone will test MAC-negative after six months with just multidrug treatment if they haven't already?

    So the chances are slim, and for that reason ARIKAYCE is my go-to treatment.

    So I think the question people have, when they look at this data is, how many people in this study stayed MAC-negative?

    So, Judy, that's a wonderful point. There's actually an additional analysis that was done to determine how many people that tested MAC-negative remained MAC-negative over time. This analysis found that after completing treatment ARIKAYCE kept working, and you can see that here.

    So, if I'm reading this right, the first bar says ARIKAYCE helped twice as many people test MAC-negative at the end of treatment.

    Correct, and if you followed the time points you'll see that three months after completing treatment, and 12 months after completing treatment, 55% and 46% of people who tested negative with ARIKAYCE, were still testing negative compared to those who took the multidrug treatment alone just at 0%.

    What about those additional study results?

    There is no improvement in the six-minute walk test, or the Saint George's respiratory questionnaire after six months of treatment.

    When I was going through this, the data is what my doctor and I used to help map my own journey.

    And I think it's very helpful.

    We've talked a lot about the different conversations you have with patients, what are the things you tell them that they may experience with ARIKAYCE?

    Something that I'd like to share with all my patients, no matter what, is the potential side effects that they may experience.

    Yes, that was one of the first questions I had when I started treatment.

    So I think everyone does, and I always tell my patients about the more serious respiratory side effects that they can experience with ARIKAYCE. So ARIKAYCE can be associated with allergic inflammation of the lungs, coughing up blood, severe breathing problems, worsening of chronic obstructive pulmonary disease or COPD, as well as more severe allergic reactions. So speaking of allergic reaction, ARIKAYCE is a type of antibiotic known as an aminoglycoside. If you're allergic to that type of drug or antibiotic or any of the ingredients, you should not use ARIKAYCE. ARIKAYCE is also associated with hearing loss or ringing in the ears, worsening kidney problems, and worsening muscle weakness.

    Do people who add ARIKAYCE experience serious side effects?

    We actually see in the study 16.1% on the multidrug treatment alone had these serious side effects compared to 19.7% of people who added ARIKAYCE.

    What about the common side effects of ARIKAYCE?

    I like to cover those, because you don't want people to be caught off guard with things like changes in their voice or hoarseness, which we see was experienced by 48% of people in the clinical study. Cough is another common one, reported most often during the first month of treatment, it was experienced by 40% of people who took ARIKAYCE. There's also muscle pain and sore throat experienced by 18% of people who took ARIKAYCE. So the one I also mentioned is increased sputum production, which happened in 6%, for people who are already symptomatic from a respiratory standpoint, it's obviously very important to mention that common symptom.

    Yeah when I was on ARIKAYCE it wasn't really the cough, but a little bit of post-nasal drip and some ear ringing.

    So everybody is different, and patients may have different side effects. And I emphasize it is important to speak with your physician about them. I don't want to hear three months from now about any side effects that one is experiencing because we want to make sure that we can intervene as early as possible.

    Do patients ever say, “Alright, now that I've started this new treatment how long until it starts working?”

    They do, and in the ARIKAYCE clinical study people who tested MAC-negative were on treatment for one to four months before they received their first negative test.

    From experience I had to ask about these things multiple times to really understand them.

    And that's OK, many people do. What I want patients to know, is that we're there for them. From a communication and a support standpoint, to help them get what they need.

    I really appreciate you sharing your knowledge and information about ARIKAYCE in particular. It's meant a lot to me, thank you.

    It's been my pleasure.

    The question I needed answered most in my treatment journey: “What's next?” I needed someone to tell me what I could experience and how to manage the side effects along the way.

    If there's one bit of advice I can offer to others, it would be to make sure they have the right doctor, who can guide them every step of the way.

    Let's see what we can learn at our next stop.

    Throughout my MAC treatment journey, I found that it's a rare but wonderful thing to have someone who really gets you, someone who can walk a mile in your shoes, and you in theirs. That someone for me is Elisse.

    Hi, this is so great –

    Oh my gosh –

    (mixed dialogue)

    Come in, come in.

    Thank you.

    So how was your trip?

    It was great, thank you again.

    Thank you for coming.

    Cheers.

    I wonder if you could share with me how it felt when you were diagnosed with MAC lung disease.

    Oh absolutely. I was shocked. Honestly, it was like a deer in headlights. My primary care doctor took an X-ray of my lungs because she didn't like what she heard. When I saw the X-rays, I could see the damage she pointed out. I already had damage to both my lungs, and there was a great sense of urgency, to get started on treatment right away.

    So you were on an initial multidrug treatment for MAC, did that work for you?

    It did for a while, I did the treatment and once I was done, the initial multidrug treatment, I was OK for a while.

    Could you tell me more about how you heard about ARIKAYCE?

    I actually heard about ARIKAYCE from my pulmonologist. I was hopeful that this would help me test MAC-negative, and it did so I was thrilled.

    When you were first diagnosed with MAC, what was your experience with your physicians?

    Well, my first experience with my infectious disease doctor, uh was not great. I got no information from them, I would ask questions get one-word answers, it wasn’t working, so I called my pulmonologist to ask to find somebody else - which he did, and it was a completely different experience with someone else. I got my questions answered, we had a rapport, I think it's very important to find a doctor that you can work with.

    Did anyone give you a bird's eye view of this situation?

    I did not have an overview of the whole thing, of where I was going to be going and how long it was going to take to get there. They did tell me that the first time, when I was MAC-positive, my treatment was going to be 12 months. And the second time I was MAC-positive, my treatment was going to be for 18 months, and that was with the ARIKAYCE.

    My experience isn't all that dissimilar. It would have been very helpful if someone had just helped me set the expectations. One of the big things that people try to manage is the side effects, how did that work for you?

    I actually had hoarseness in my voice a few times and I actually lost my voice twice for a few days each time.

    Mine were very similar. I also lost my voice, I kind of expected it. I had gone through the side effects with my doctor, I had a little bit of nasal drip, even like the blogs and the forms I was on, it was helpful in trying to manage all of that. I know that this can be a long journey, what would be your best advice to people with a diagnosis of MAC lung disease?

    I would advise them to remember the doctor is there for you and your health, don't be afraid to ask questions.

    Meeting up with other people, someone like Elisse, who also has NTM MAC lung disease, and has traveled the same path with me, helps a person not feel alone. You're not alone on this island. Not only with finding ARIKAYCE, but also finding other tools and other support such as good physicians, as a good team that you're working with.

    We all know rare diseases can be lonely diseases, but they don't have to be. Here's what I wish someone would have told me: Remember to advocate and ask all your questions, it's a long journey but there’s support to help you persevere. I always say, the best way to get answers is to have honest conversations. On this quest for knowledge I saw the care that went into designing ARIKAYCE. I learned having the right doctor for you helps you on your treatment journey, and a friend reminded me, I'm not dealing with this disease alone.

    To the patients watching, stay hopeful. Yours truly, Judy.

  • Adding ARIKAYCE helped people test MAC-negative. See the study results
  • Stay up-to-date with MAC and ARIKAYCE information
Next: ARIKAYCE support
download icon

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

ARIKAYCE can cause serious side effects, including:

  • allergic inflammation of the lungs. These respiratory problems may be symptoms of allergic inflammation of the lungs and often come with fever, wheezing, coughing, shortness of breath, and fast breathing
  • coughing up of blood (hemoptysis). Coughing up blood is a serious and common side effect of ARIKAYCE
  • severe breathing problems. Severe breathing problems can be symptoms of bronchospasm. Bronchospasm is a serious and common side effect of ARIKAYCE. Bronchospasm symptoms include shortness of breath, difficult or labored breathing, wheezing, and coughing or chest tightness
  • worsening of chronic obstructive pulmonary disease (COPD). This is a serious and common side effect of ARIKAYCE
  • serious allergic reactions. Serious allergic reactions that may lead to death have happened to people who take ARIKAYCE. Stop taking ARIKAYCE right away and get emergency medical help if you have any of the following symptoms of a serious allergic reaction: hives, itching, redness or blushing of the skin (flushing), swollen lips, tongue or throat, trouble breathing or wheezing, shortness of breath, noisy high-pitched breathing (stridor), cough, nausea, vomiting, diarrhea, feel cramps in your stomach area, fast heart rate, feeling light headed, feeling faint, loss of control of the bowels or bladder (incontinence), and dizziness

While using ARIKAYCE, these side effects may become serious enough that treatment in a hospital is needed. Call your healthcare provider or get medical help right away if you have any of these serious side effects while taking ARIKAYCE. Your healthcare provider may ask you to stop using ARIKAYCE for a short period of time or completely stop using ARIKAYCE.

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

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

  • have asthma, COPD, shortness of breath, or wheezing (bronchospasm)
  • have been told you have poor lung function
  • have hearing problems, such as ringing in your ears or hearing loss
  • have dizziness or a sense of the room spinning
  • have kidney problems
  • have neuromuscular disease, such as myasthenia gravis
  • are pregnant or plan to become pregnant. It is not known if ARIKAYCE can harm your unborn baby. ARIKAYCE is in a class of medicines that may be connected with complete deafness in babies at birth. The deafness affects both ears and cannot be changed
  • are breastfeeding or plan to breastfeed. It is not known if the medicine in ARIKAYCE passes into your breast milk and if it can harm your baby. Talk to your healthcare provider about the best way to feed your baby during treatment with ARIKAYCE

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

ARIKAYCE may cause serious side effects, including:

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

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

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

What is ARIKAYCE?

ARIKAYCE is 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.