Find out if ARIKAYCE is right for you

On treatment but still testing positive for MAC?

  • MAC can be hard to treat. If you are still testing positive for MAC, you are not alone. For 1 in 3 people, initial multidrug treatment may simply not be enough to test MAC-negative. That’s where ARIKAYCE comes in.

Treatment experts strongly recommend adding ARIKAYCE

The NTM Treatment Guidelines,* created by leading scientific communities, recommend adding ARIKAYCE, the first and only FDA-approved treatment for adults still testing positive for MAC after 6 months on an initial multidrug treatment.

The NTM Treatment Guidelines recommend:

When MAC is diagnosed: Start a multidrug treatment

The guidelines suggest beginning treatment rather than waiting, especially for those with:

  • a more severe infection
  • a compromised immune system
  • severe fatigue or other signs of decreased quality of life
  • cavitary disease

Treating MAC lung disease involves taking more than 1 medication. These medications work together to fight the MAC bacteria.

During Months 1–5 of treatment: Frequent sputum tests

The guidelines recommend regular doctor visits and monthly sputum cultures to assess if a person is responding to therapy.

6 months after start of treatment: Test MAC-negative?


The guidelines recommend that doctors evaluate if the multidrug treatment is working after 6 months.

NO. Still MAC-positive

Treatment guidelines strongly recommend adding ARIKAYCE when continuing to test positive after 6 months of treatment.

ARIKAYCE was approved by FDA using the Limited Population pathway for a limited and specific patient population.

YES. Tested MAC-negative

Continue multidrug treatment.

Additional recommendation: Continue on treatment for 12 months after testing MAC-negative

The guidelines recommend staying on NTM treatment for a minimum of 12 months after testing negative for MAC. This is to help make sure the MAC bacteria is cleared from your lungs.

ATS/ERS/ESCMID/IDSA NTM Guidelines.
ATS=American Thoracic Society; ERS=European Respiratory Society; ESCMID=European Society of Clinical Microbiology and Infectious Diseases; IDSA=Infectious Diseases Society of America; MAC=Mycobacterium avium complex; NTM=nontuberculous mycobacteria.

See what these MAC lung disease specialists are saying about ARIKAYCE

Drs. Philley and Drummond discuss their experiences with MAC lung disease and prescribing ARIKAYCE.

Transcript

Mycobacterium avium complex, or MAC, is the most common form of nontuberculous mycobacteria.

You may be put on the guideline-recommended multidrug therapy, which means that you will take more than one medication at the same time.

Patients with MAC lung disease are treated with more than one antibiotic because unfortunately, there isn't one single antibiotic that can effectively kill the bacteria.

These medications work together to attack the NTM bacteria in different ways. Using more than one treatment also helps prevent the bacteria from becoming resistant to one antibiotic.

Unfortunately, standard combination therapy alone may not work for every case. ARIKAYCE was studied and approved by the FDA for MAC lung disease patients who did not respond to combination antibiotic therapy alone.

ARIKAYCE is added to combination therapy and is not taken alone. The FDA approved ARIKAYCE under a Limited Population pathway, which is for a limited and specific patient population. ARIKAYCE is an inhaled antibiotic that you take using a nebulizer.

It's different from other inhaled antibiotics because the amikacin is contained inside tiny particles called liposomes.

These liposomes are designed to help the medication reach the MAC bacteria in the lungs.

For standard MAC treatment based on guidelines, I typically plan to maintain patients on treatment for 12 months of negative sputums beyond initial culture conversion.

I also apply this to ARIKAYCE treatment. In the clinical trial, the protocol called for 12 months of treatment after the first month of culture conversion.

When patients first begin ARIKAYCE, I usually spend time talking with them about how the device works, specifically the nebulizer, from which they will inhale the drug.

We also discuss the adverse effects that may or may not occur with the drug and when to notify their physician, and we also discuss how to follow up as far as routine clinical monitoring and setting expectations about appointments.

Everyone responds differently to treatment. If you have questions about the possible side effects associated with ARIKAYCE, be sure to speak with your doctor.

ARIKAYCE is associated with a risk of increased respiratory adverse reactions including allergic inflammation of the lungs, coughing up blood, severe breathing problems, and even worsening of COPD.

While using ARIKAYCE, side effects may become serious enough, such that treatment in the hospital is required. Call your healthcare provider right away or obtain medical help if you are experiencing any serious side effects while taking ARIKAYCE.

So, I explain to the patient the goals of therapy and the length of therapy which is 12 months of negative sputum cultures. When you're on treatment for MAC lung disease, it's important to check sputum cultures frequently to see if MAC continues to grow in the culture.

That involves coughing up a sputum sample, and often either taking it or mailing it to your doctor's office.

While patients are taking ARIKAYCE, follow-up appointments are necessary with providers. At those appointments, we usually check sputum cultures to understand if MAC is still growing in the sputum.

We also talk about the treatment response to ARIKAYCE and any possible adverse effects the patient may or may not be experiencing. In addition, radiographs may or may not be necessary as well as other testing as defined by the doctor.

I usually see a patient within the first month after starting a new therapy, and then every three months thereafter. Three months is a good time point to monitor these patients because I'm also doing routine audiograms while on treatment.

Recently, in clinic, I saw a 67-year-old white female. I placed her on a standard oral multidrug regimen, and she tolerated the regimen quite well.

We checked sputums monthly and, despite the use of a correct regimen, she still remained culture-positive for MAC at six months.

I placed her on daily ARIKAYCE, according to the package insert, and several months later, her sputum converted to negative. And she continues to do well on this multidrug regimen. She was very pleased with her progress and, of course, so was I.

You can learn about how many people in the ARIKAYCE clinical study achieved culture conversion, the side effects they experienced, the sample size, types of patients enrolled, and how many patients successfully completed the study.

You can learn more about this on ARIKAYCE.com.

Lynn

“I was put on a multidrug treatment and I thought that’d be the answer. To find out it wasn’t working was incredibly disheartening, frustrating, and a little bit scary. Then I found ARIKAYCE.”

Judy, a real ARIKAYCE patient

In the Know Program: Journey to ARIKAYCE

Watch MAC lung disease expert Dr. Gwenn Huitt and Judy, a real ARIKAYCE patient, discuss topics that can help you learn more about MAC and its treatment options, including adding ARIKAYCE.

Transcript

Hi everyone, and welcome to today's program. Thank you for joining us for In The Know, let's talk about ARIKAYCE. Today's program will focus on the journey to ARIKAYCE, amikacin liposome inhalation suspension, and you'll hear from a leading physician expert and a patient who is taking ARIKAYCE. Before we begin, I'd like to share some important information you should know about 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 or regimen. 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. 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.

Now I'd like to introduce Dr. Gwen Huitt, who will lead today's discussion. Dr. Huitt is a physician with 29 years of experience specializing in Infectious Disease at National Jewish Medical and Research Center, and NTM Center of Excellence. She's also a professor in the Department of Medicine, Director of The Adult Infectious Disease Unit, Chair of the Medical Executive Committee and Chair of the Mycobacterial Consult Service at the National Jewish Health in Denver, Colorado. Her research mainly focuses on infectious disease, pneumonia and germ theory of disease. Dr. Huitt is a distinguished physician, having published many articles on NTM, she is also the recipient of many awards, such as America's Top doctor for Castle Connolly Medical Limited. We are delighted and honored to have her here with us as our expert for this program. Over to you, Dr. Huitt.

Well, thank you so much, Nicole. That's a very generous introduction, and I'm really excited to be able to participate in the program, and I will not be the only person speaking during this program, I've got a great colleague joining me, and Judy will really do a very important service for everyone in presenting a patient's perspective of the use of ARIKAYCE in her particular situation. So Judy, if you could introduce yourself and tell us a little bit about yourself.

Sure, thank you, Dr. Huitt. I really appreciate that. Hi everyone. I'm Judy, my husband Mike and I live here in Wisconsin most of the time at our central home. Part of the time we escape the frozen tundra down to Florida. I have worked in the printing industry, starting off actually as a trades operator, as a press operator, dark room, eventually going into sales and sales support. We have 10 wonderful grandchildren, of course, and we're having a great time watching them grow up. I'm looking forward to sharing my story today. Thank you. Back to you, Dr. Huitt.

Great, thanks so much. And I think we're just gonna have a wonderful program and I'm really excited to go forward today. So we do have a packed agenda, and you're all tuning into this because we really wanna focus on the journey to using ARIKAYCE. And during this program, we are going to cover several topics including understanding MAC lung disease, getting started on a multi-drug regimen and how that goes, as well as an overview of when and how we would add ARIKAYCE's treatment and where it might fit in with a particular treatment regimen in a patient that has pulmonary MAC infection. And last but not least, certainly, we're going to give you a few tips and kind of hints about how you might talk to your doctor, how you might approach your own underlying lung disease and infection with pulmonary MAC and just move forward and empower yourself in treatment regarding pulmonary MAC. So let's go ahead and get started. Well, what is MAC lung disease? And who might be at risk for having MAC lung disease?

Well, I think the first thing I always like to tell patients is, there are varying degrees of MAC lung disease, and so every patient is not created equal, and every disease process associated with MAC lung disease may be different, but we need to take it very seriously, because if we don't take it seriously and if we don't address it regarding treatment in particular, and management in the proper timeframe, it can really progress and cause further lung damage over time, if we leave it untreated. NTM lung disease and MAC lung disease in particular is caused by a bacteria that really we find everywhere in the environment, it's particularly a water and dirt-born organism, so we all encounter it all the time, but interestingly, the MAC germs are the most common form of non-tuberculous mycobacteria organisms, and they comprise about 80% of cases of MAC lung disease.

Well, if we're all exposed to it all the time, how come some people MAC, a lung disease and others don't? Well, the honest answer is, we don't know that yet, we're constantly exposed to it. In some of the areas that we're exposed to MAC in the environment would be tap water sources such as showers or perhaps drinking water, shower heads, mist from the misters in the grocery store or out in restaurants when you're sitting under those misters, yeah, you're inhaling a lot of mycobacteria and a lot of that might be MAC. And then if you're a big gardener and you're breathing in dust and dirt outside, we know that MAC is in these sources as well, so really we're constantly based in MAC from environmental exposure. But if we're all exposed all the time, the question I commonly get from patients is, "Well, if we're all exposed, why am I the lucky one who got MAC lung disease?"

Well, we know that there are certain conditions that really predispose a patient to coming down with a more serious form of MAC infection that will lead to MAC disease, and a few of those include bronchiectasis, and we know that about 50% of people with bronchiectasis may actually have active lung disease, and in those patients, MAC lung disease is the most common form of bacteria, or MAC is the most common group of bacteria that affect their lungs. An additional group of patients that are quite vulnerable to a MAC lung infection would be those patients with underlying COPD or Chronic Obstructive Lung Disease, and we know that COPD patients may be up to 16 times more likely to get NTM lung disease because they have underlying structural lung problems just like patients with bronchiectasis, that once these germs repeatedly gain access to the lungs, they can set up shop and cause problems. Additional patient populations that might be vulnerable to MAC lung disease would be those with underlying asthma and recurrent pneumonias.

Additionally, we've known for decades that certain groups of people may be more vulnerable to acquiring MAC and then progressing to lung disease associated with MAC, and in the old days, and I'm old, we used to term this the Lady Windermere syndrome, and the common two groups of patients that we would see would be a smoker, usually a male who was a smoker, that was one group, and then the much more prevalent population was women who were tall and were thin and usually were in their 60s, 70s of age group, they tended to live in coastal areas, particularly in the United States, but we now know it's certainly not limited to those patients in coastal areas. Anyone in the middle part of the United States can certainly acquire MAC as well, but we do see it more commonly along the coast, and some people do have weakened immune systems as well.

Well, what are some of the signs and symptoms if you think you might have MAC lung disease? Well, it's interesting because the MAC lung disease symptoms may really mimic many other diseases and disease processes, so you may have a chronic cough, well, people with COPD often have a chronic cough, rarely DOEs, and so it differs from asthma in that regard. Generally, you're not experiencing wheezing like asthmatics, but asthmatics cough a lot as well. It mimics other disease processes, and because of that, your practitioner, your general practitioner, and turnus family practitioner may be looking in other directions, so it may be a little difficult to diagnose initially. So the common theme I hear from a lot of patients is, "Gosh, it took me years to finally get to the right place and to the right doctor to get diagnosed."

So it's important and empowering, and part of our job today is to empower you as a patient to say, "Well, what are the common symptoms that somebody with MAC lung disease might be experiencing?" And generally speaking, chronic cough, fatigue and shortness of breath are really the common themes. Now layered on top of that might be sputum production and commonly is sputum production. But the rare person just coughs and has shortness of breath and really doesn't produce any sputum, and when these just are relentless and continue to go on despite your doctor prescribing umpteen courses of antibiotics or trying on an antihistamine for allergies or giving you a breathing treatment. If nothing's working, then it's time to look under other rocks and start looking to think that it might actually be MAC Lung disease. Well, I'd like Judy's impression right now what her journey was when she started on this process. If you don't mind, Judy?

No, I don't mind at all. As a matter of fact, I think you described my situation almost to a T. I had been diagnosed 12 plus years ago, but it was a long time getting there, I had... And it seemed like analyst rounds of pneumonia every time I went in vacation, I get sick about the third day and I'd be running a temperature. It's extreme fatigue. I'd lose my appetite, which is not my nature. And my physician kept looking at it and the history of all the different rounds of pneumonia and being sick on a regular basis like that, and she thought maybe we ought to dig a little deeper on the situation, it actually came to a head one night, I was coughing so much and I could not stop coughing and I cracked a rib and I was in so much pain, I didn't realize where that pain was coming from.

My husband took me to the emergency room, and I think that that coming to a head at that point just made my internist look at it harder and get me over to a specialist. It started with a pulmonary physician who ran me through a whole series of tests, of course, including a bronchoscopy, which finally led to the diagnosis of MAC lung disease, and they referred me to an infectious disease specialist at that point. Back to you, Dr Huitt.

Great. Thanks, Judy. And I would just reiterate that this seems like a story that I've literally heard thousands of times. Unfortunately, including the cracked rib portion of the story. I wouldn't say I've had thousands of patients with cracked ribs, but I've certainly had hundreds unfortunately, and because women are women and have thinner bones, may have a little bit of frail bones like osteopenia or osteoporosis, so fragile bones, and they start having a racking cough this can really lead to some problems. I've even had patients with cracked ribs collapse their lungs, so this really can be complicated, and thank you so much for sharing that because I think it's so important for all of the patients that are out there listening to this to recognize that they're not alone, and that if they're experiencing some of these same symptoms, gosh, it seems like this is something that needs to be assessed by their docs, see if they do have NTM lung disease.

Yes.

Well, Judy just described a little synopsis of how her doctors proceeded with diagnosis, and ultimately we'll talk about treatment of NTM lung disease, but I think it's important... Again, the theme is empowering you as the patient to kinda push us physicians to help you and help you sooner than later, not have to go 10 years, not even have to go five years to make a diagnosis, so if you think you have these symptoms, you've failed multiple courses of antibiotics, you still have this chronic cough, then think that you may have MAC lung disease and talk to your physician about getting tested. Now, in most cases, I would say you're talking to your primary care doc first and foremost. This is going on, they've been trying the best they can, obviously, everyone at some point in time says uncle and I'm gonna have to pass you on to a specialist, which I would say in most cases, before the diagnosis is actually made, patients get referred to a pulmonologist.

And it's important to make this referral because the longer this disease process is not addressed and treated, you are likely to have more permanent lung damage that is gonna lead to worsening bronchiectasis and structural problems, which will make it much more difficult to treat. So some of the things that you can say are illustrated in the slide, just say, "Doc, we've been trying to deal with this for months or years, can you please refer me to a pulmonologist or infectious disease physician?" So empower yourself to have those services at hand. And so what a pulmonologist is generally gonna do, obviously they're gonna take a history in physical, they're going to get an x-ray and hopefully they're going to get... In addition to a chest X-ray, they're gonna get a CAT scan of your lungs, because the chest x-ray just does not diagnose bronchiectasis very well.

So we really need to go the extra step and see, "Gosh, has there been damage that's already been done," and we can see it visually on the CAT scan picture. Then they need to collect some sputum or something from your lungs, some secretions, so that we can send that to the laboratory and see if they are actually able to grow MAC in the lab, and unfortunately, that takes about, I don't know, six to 10 weeks depending on the lab, and I think that's the hardest thing for patients and physicians, is the length of time that it takes to culture this germ in the lab. But in the meantime, your doctor can do several things additionally, like help you learn how to keep your lungs cleaned out using devices that vibrate your lung and you can at least if you produce mucus and stuff, you can clear your lungs out while you're waiting to start treatment.

And after the six to eight to 10 weeks and your lab calls your doc back and says, "Listen, yeah, we're definitely growing MAC," then the time comes where you and your doc have to talk about, "Okay, we appear to have diagnosed me with MAC, what do we do?" Well, the standard regimen that is used is to use a multi-drug antibiotic treatment approach, and that's usually called multi-drug therapy, and usually it consists of three different antibiotics at the same time. And the reason we use three medicines at least is three is better than two, but theoretically, two would be okay, but three is standard of care, is that we want to attack this bacteria from multiple directions so that the germ does not get smart to it and doesn't develop resistance to any one of the three antibiotics.

So we're sort of confusing the germ, so that it can't develop resistance. And that's very, very important because I would say from a physician standpoint, one of the problems that we see a lot is that doctors may just give you one antibiotic at a time, and yes, it helps you initially, but unfortunately, when you test the germ again two months later after you've been on only one medicine, that germ is now resistant to that one antibiotic, so we can't use it anymore. So it just lessens the choices that we have in a treatment regimen. Well, luckily, physicians have a guideline, they have a document that was revised in 2020, and what this document does is it helps doctors know how they should approach not only the diagnosis of NTM lung disease, not specifically MAC, it talks about many of the non-tuberculous mycobacteria, but how do you test and then what are the recommendations for treatments and for monitoring, if you do decide to go on treatment and you and your doctors feel that that's the right way to go.

This document was developed and published by... Gosh, about 20 physicians and scientists who are in the field of NTM lung disease, and we're very lucky to have had an updated document in 2020 to help physicians. And patients have access to this document too. You can just look it up on the internet. So it's available to anyone. Well, the guidelines recommend starting multi-drug therapy rather than watchful waiting. For the most part, especially if you have evidence of severe infection. And what is severe? Gosh, you have a lot of symptoms, you're coughing, you're fatigued, you might be losing some weight. Let's say you have a compromised immune system, but you're plagued by these symptoms and radio-graphically what we would look at is, gosh, you've got evidence of bronchiectasis now, or in the worst case scenario, you've got something called a cavity, meaning a hole in your lung.

Well, this all indicates more aggressive MAC disease and you better start on medicine immediately. Antibiotics meaning at least three drugs, and not just try simple things. It's time to get started on treatment. Well, the guidelines also recommend that you go to see your physician very frequently, and once they start you on a treatment regimen with the multi-drug antibiotics, they need to check your sputum cultures usually every month or at the most, every two months, because we wanna see if the antibiotics are actually working. I mean, you're going to be able to tell your doctor, "Gosh, yes, I'm noticing a decrease in my cough since I've started antibiotics and I'm having less fatigue." So that's one thing we certainly wanna watch as well, but we wanna see what your sputum cultures are doing, and we'll get to it later, but we're also going to be checking some blood tests on you periodically to make sure that you're not experiencing any toxic side effects from any of the medicines that we have you on.

Well, a lot of patients say, "Well, if I'm gonna embark on treatment, how long is that going to be?" Well, the guidelines say, we want to for sure be watching your cultures and have 12 months of negative cultures. So the clock starts ticking the first time you get a sputum that is culture negative, and we try to go for 12 months after the first culture negative 'cause we wanna be sure that we have killed the bacteria. And the multi-drug regimen actually works for most people, but there is a proportion of people that it doesn't work for, and we have to advance the drug regimen and increase the number of antibiotics that a patient is taking and perhaps consider additional modalities rather than just pills.

So what the new NTM guidelines, the 2020 NTM guidelines strongly recommend, is that adding this new nebulized inhaled antibiotic ARIKAYCE to the underlying multi-drug regimen that you've already been taking, if you continue to test positive after taking the three pills for six months and you still haven't gotten one culture negative sputum. So that's really important. And once you reach this point in your treatment course, there's a lot of discussion that needs to be undertaken with you and your physician, and particularly, we wanna often talk about potential side effects and what to expect when you're taking that. So, Judy, can you share with us what you and your physician spoke about when you got to this point?

Sure. Once I had the diagnosis for MAC... Thank you, Dr. Huitt, by the way. I was immediately put on the multi-drug regimen because I was getting sicker, I almost felt like I was fading away, which is not my nature, I'm a active person. And I really wanted to take an aggressive approach to it, I wanted to knock it out of my system. I felt like if I had gotten this diagnosis earlier in the program, I wouldn't have had the lung damage or possible permanent lung damage that I might have experienced at that time. And going on the multi-drug regimen, I thought that was the answer. I did some research and I also saw that that was really the protocol for the MAC lung disease. And two years ago, three years ago, when I realized that there were multiple re-occurrences and the multi-drug regimen just wasn't going to cut it any longer, it was not a good thing. I was disappointed, it was disheartening, and frankly, it was a little scary to find out that the drug regimen that I'd been using for that amount of time was not really working for me.

So we started to look at alternatives, I had talked to my doctor about ARIKAYCE, where I found a product sheet on a community website, I found some support there. I was looking, kind of searching and researching for myself to see what else might be out there. And I found ARIKAYCE, and I found the product sheet and I took it into my infectious disease specialist. She took a look at it, we talked about it, and she thought like I did, it might be another tool in the toolbox, something he could add into my regimen that might provide a hopeful answer for me. And when it came to the point to make that decision, she helped me find the research tools to be comfortable with the process and to think of the ARIKAYCE as another tool in my toolkit that I could apply to my lungs. So it was a journey, but a successful journey here, getting to the point where we were introduced to ARIKAYCE. Thank you, Dr. Huitt, back to you.

Sure. Well, I think that that is very... Fortunately, that's very typical in what I hear patients that come and see me is they've had the opportunity to look on the internet, there's so much information that's out there. Compared to 20 years ago, it's amazing. So if you have access to internet or a library, if you don't have a computer at home, by all means, try to go and educate yourself. Because honestly, there are a few docs that still need educated regarding this product and how to approach NTM lung disease, 'cause not everyone is as well-versed as they should be. So once again, I think my job here is to empower you, the patient, to, as Judy said, have more tools in your toolbox to be an advocate for yourself and get the best treatment possible. And certainly, what the latest guidelines are showing patients should do.

So let's launch into the antibiotic itself, what actually is this antibiotic ARIKAYCE that we talk about? Well, I will tell you, I've been doing this for 30 years and we have never had an FDA approved, and you've heard a lot about FDA approval lately with the current COVID-19 pandemic. So FDA is our government body that diligently looks at any new medication that's coming to market in the United States. So ARIKAYCE is the first and only FDA-approved treatment option that's been designed specifically for adults who do not respond to a multi-drug regimen alone for six months and have continued positive cultures for MAC lung disease. And again, the important thing to always remember about this drug is that this antibiotic really needs to be added onto an existing multi-drug regimen therapy, so you would never take ARIKAYCE by itself, it's an add-on antibiotic. But an important add-on antibiotic, most certainly.

Well, it's also important when we start any medication, we wanna know about the potential downsides to this that we as the patient may need to watch for, and certainly we as the prescribing physician needs to know exists so that you can have this dialogue with your patient. So, ARIKAYCE can cause serious side effects that include: An allergic inflammation of the lungs, it can also cause coughing up of blood, and we refer to this as hemolysis. It can also cause severe breathing problems, can also cause worsening of COPD or Chronic Obstructive Pulmonary Disease, and has also been known to cause serious allergic reaction. 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, and that's so important. And also, last but not least, do not use ARIKAYCE if you know that you have an allergy to any aminoglycoside or any of the ingredients that are contained in ARIKAYCE.

So, how do you take ARIKAYCE? Well, it's rather novel actually, it's a liquid and it's an inhaled antibiotic that you take using a nebulizer that's pictured on this slide. It's different from other inhaled antibiotics because it's active ingredient, known as amikacin, is contained inside tiny particles called liposomes. And when you inhale the ARIKAYCE medicine, these liposomes contain the medicine, and they then travel into the lungs to get inside the airway to where this MAC germ is hanging out. And once they're inside of the lungs, they release the medicine to fight the MAC bacteria at the site of infection. And you'll see there's a code on this slide that you can use your smartphone on to see how ARIKAYCE works, it's a video that you can watch at the end of our program if you'd like. But there's an illustration on this slide showing the little amikacin, which is the active ingredient, the antibiotic molecules inside the yellow core of the liposome.

And a clinical study was performed, as with any new drug or new antibiotic, certainly. And this looked at 224 people, and it was ARIKAYCE plus the multi-drug regimen, and it was in people with MAC lung disease who either stopped responding or never responded to their previous treatment. And the key goal was, were we able to culture convert these patients, meaning did the sputum ever go to culture negative by month six? Additional measures that were looked at in this study included: Improvement in the distance walked after six minutes, and improvement in overall health, daily life and perceived well-being in patients with lung disease. And that parameter was a questionnaire. Well, what was important in this study is adding ARIKAYCE clearly helped patients test MAC negative for 12 months and beyond, and as illustrated here, the primary results showed that ARIKAYCE helped three times as many patients test MAC negative at six months or of the 224 patients, 65 tested negative versus those patients who were not taking ARIKAYCE, only 10 of 112 tested MAC negative.

Then when we got to the 12-month time period, which is the end of treatment, ARIKAYCE helped seven times as many patients test MAC negative at 12 months. Or in the initial 224, there were 41 patients who continued to test negative in the ARIKAYCE group and only three of 112 in the non-ARIKAYCE were testing negative. And then most importantly, I would say, they tested for three additional months after all antibiotics were off and looking to see whether sputum cultures converted back to positive and only in the ARIKAYCE patients who had tested negative, and then we continued to watch them, all of them, three months after all drugs were stopped, only the patients that had been exposed to ARIKAYCE were MAC negative (16%). So 36 out of 224 and absolutely zero of the patients who were on the multi-drug regimen alone, so that's a very significant finding that was ascertained from this study. And for those who successfully tested MAC negative, ARIKAYCE also helped more patients stay that way.

And as you can see, at the end of treatment of using ARIKAYCE in the regimen, ARIKAYCE helped twice as many patients test MAC negative at 12 months as opposed to those patients in the gray boxes who had not received ARIKAYCE. And again, only ARIKAYCE patients, the patients that we're exposed to ARIKAYCE, once all antibiotics were stopped, were able to be culture negative after three months. So none of the patients who never received ARIKAYCE and 36 out of 65 receiving the ARIKAYCE were able to stay culture negative three months after stopping all medicine. So once again, this is a very important point that ARIKAYCE really did provide a significant benefit if it was used in your treatment regimen. In this clinical study, they also, as I said, looked at whether or not you improved with a six-minute walk test, and there was none on the six-minute walk test and the St. George's Respiratory Questionnaire measurements at the end of six months also showed no improvement in the ARIKAYCE versus the non-ARIKAYCE group. Judy, you certainly had experience with this. And when your doctor and you talked about adding ARIKAYCE, what was your experience once you added ARIKAYCE?

Thank you, Dr. Huitt. We made the decision to add in ARIKAYCE into the toolbox, and I worked with a very collaborative team. I had an infectious disease specialist at the center of my team, I worked with a pulmonary specialist, pulmonary technicians to measure my process and my progress as we went along at each stage of the game, and I felt that there was an opportunity with ARIKAYCE to make that type of progress that I really wanted. I spoke with my infectious disease doctor at length about different clinical trials, about the supporting evidence that would help me make good decisions about how I worked with ARIKAYCE and what it could really do for me, so we discussed that at length.

And it felt like it was a hopeful tool for me, it was something that might provide an answer. I just thought, why not give it a shot? I certainly needed something different after all the wrongs that I had been through previously. I've been very happy with that decision to move forward, I've had two negative sputums, I'm pretty happy to report that. And I have a third culturing currently, my infectious disease specialist and I did a little happy dance. I was one of her first patients when she came on board at the particular medical society, a group that I work with, and she knew how diligent I was about working towards this goal. So it was pretty cool, we thought... We kind of think we might have had our last appointment, let's see what happens. So thank you, Dr. Huitt.

Well, that's a wonderful story. And I will reiterate that I've heard similar stories from my own patients on many occasions, and I have done a happy dance with some of my patients as well, so I think that's a common theme here. Well, the next phase I think that we wanna talk about is how do you talk to your doctor, and how does your doctor talk to you about ARIKAYCE? And I again, want to have the theme that we are trying to empower you to educate you as the patient, so that sometimes you can educate your physician, you can be their teacher. But know the important questions to ask once you're at this point, when you're thinking about ARIKAYCE and adding it to your already failing multi-drug regimen. So typical things that you would ask is, "Gosh, how do I know whether ARIKAYCE is working for me? What kind of monitoring are you gonna be doing on me? Well, do you think ARIKAYCE would be a good option for me? How long am I gonna take the treatment? And how are you gonna monitor me? Not only with sputums, are gonna get CAT scans? Do I have a log book that I... "

Just all these kind of questions that you and your doc sorta get your plan sorted out so that no one is confused about what you're gonna be doing going forward, and quite honestly, this discussion should happen even if you weren't taking ARIKAYCE. Any medicine that we're taking, we should ask our doctor, "What are you trying to do with this medicine, and how are you gonna monitor me?" So the same thing is applicable with ARIKAYCE. And last but not least, I always wanna talk about potential side effects. So you wanna know the good, but we need to know the potential bad, 'cause everything's got a potential dark side. So your doc and you need to know that ARIKAYCE may cause some serious side effects that include, but are not limited to hearing loss or ringing in the ears, and we commonly refer to that as ototoxicity.

Some people may say... Obviously, you know what hearing loss is. But tinnitus, ear buzzing, whooshing of the ears, ear fullness, like you need to pop your ears. So you have to watch for that. We have to watch for worsening kidney problems, or that's referred to as nephrotoxicity, and that's tested by a blood test called a creatine, generally speaking. So you'll be having routine blood monitoring, and you should have been doing this anyway with your baseline medication. And then in some cases, a worsening muscle weakness may occur, and this is particularly in people with underlying neuromuscular diseases. The common one that people think about is myasthenia gravis, so those are serious side effects that we really have to watch you for.

Now, the important thing is, in the clinical study, we noticed that a relatively high percentage of patients that were just on the baseline regimen, 16%, in fact, 16.1% of patients taking just a multi-drug regimen had some serious side effects, and that increased to 19.7% in patients who had ARIKAYCE added into the regimens. And then other side effects, these are the common side effects, and I'm certainly not gonna go through this whole slide, but I would say bar none, cough, when you're dosing yourself, when you're nebulizing or after you complete your nebulization with ARIKAYCE, is by far the most common theme and hoarseness, I would say would be the second thing that patients complain about. And particularly, this seems to be really common in the first month, but seems to subside, sometimes with a little modification and we'll go through that, but I would say those are the most common things that I've experienced in hearing from my patients that they have experienced it.

And in a clinical study that was done, most patients who experience side effects did not require stopping treatment, they didn't want to stop treatment because they weren't serious enough side effects that they felt like they needed to stop treatment, and 81% of patients taking ARIKAYCE along with their multi-drug therapy regimen did not stop treatment even though they had at least one side effect, and I would say my practice definitely would mirror this. But these are not the only possible side effects that can occur with ARIKAYCE, and so it's really important that you call your doctor or pharmacist for medical advice about side effects if they occur, and you may also report these side effects to the FDA, and the number is 1-800-FDA-1088.

So that's important to remember that you have that outlet to also report to the FDA. But certainly, the thing that you can do as a patient as well, is be sure and tell your healthcare provider about all the medicines that you take, not only the prescription medications that a doctor is prescribing, but any over-the-counter medications or herbal supplements or vitamins, and I'm always shocked, often, at how many of these patients don't report... How many patients don't report that to us, and it's really important 'cause there can be significant drug-drug interactions in something that you would not necessarily think would have a drug-drug interaction. So, two academic centers did a study, via phone, with 26 patients that had been prescribed ARIKAYCE during a two-month period, and this was in the United States, and this...

Insmed was not involved with the conceptualization, development, conduct or analysis of this study, and it's not included in the ARIKAYCE full prescribing information, but they provide writing assistance when the authors were publishing this, through a grant through Insmed incorporated, and what they did is they tabulated the side effects that patients were reporting and what strategies did patients and physicians use when these side effects occurred, and so, when they looked at increased coughing... As you can see, there's some bullet points going down, so oftentimes, they'll give you an albuterol treatment. Sometimes just changing the ARIKAYCE nebulize treatment to the evening. Sometimes just maybe giving it only three days a week instead of seven days a week until your vocal cords and lungs settle down a little bit, sometimes you give cough syrup or have you suck on some lozenges.

Warm salt water, commonly a gargle was very helpful to my patients, I would say, then the next group is the hoarseness or dysphonia, as we call it, and I've only had one patient totally lose their voice and all others of the myriad of patients that I've started ARIKAYCE on, I would say to a person, they develop some hoarseness and it was more significant at the beginning of the treatment. As treatment went on, the hoarseness improved over time, but again, we kinda use the same things for hoarseness with the exception of we don't give an inhaler medicine to help with that. Now, some people do develop shortness of breath or what we say dyspnea, I wanna stop here and say, if you experience anything, you should let your physician know what is going on because they're your partner in this and they have solutions that will help you, but you need to let... We can't help you, or do something about it, if we don't know about it. So back to dyspnea or shortness of breath, they might use an inhaler or Bronco dilator. We might just stop the ARIKAYCE for a few days or a couple of weeks and see if things quiet down and then restart it, maybe slow.

Maybe introducing it, not on a daily basis, but just maybe three days a week for a week, and then increase it to four days and gradually get up to full dosing again. If you're short of breath, we want you to limit your physical activity until you're not short of breath, and in people that are already on oxygen, we may sometimes say, gosh, turn up your oxygen flow from two liters to three liters and see if that will help, and then almost to a person too, I would say when they started the nebulizer treatments, they noticed more sputum would come up during the day because any nebulizer antibiotic is slightly irritating to the airways, and so that does tend to increase your volume, but it's not unmanageable in any circumstance. So, I'd like to toss it back to Judy and see if any of these things occurred in her, and did she have good options for mitigating these symptoms?

Thank you, Dr. Huitt. Yes, I did have a little bit of ear ringing, a little bit of nasal drip, I kind of lost my voice, was very hoarse for about a week, which my husband was a little inappropriately happy about... So, some site effects have benefits, but I really knew what to expect, I had a good... As I mentioned earlier, I had a really good collaborative team involved in my diagnosis and in my treatment. You made a great point about talking with your pharmacist. They were a great resource for me also, and I knew what to expect. I was prepared for it. So, nothing was a surprise to me. I kept a lot of notes and I kept referring back to his notes and I just kept... I had really committed to it, I wanted to make sure I kept after it, and I gave myself the opportunity to really let this ARIKAYCE work for me, and the side effects, they were very manageable, so it was all good. Thank you, Dr. Huitt, back to you.

One of the other things I tell my patients or ask them and plead with them to do is keep a log. So keep a notebook with you, and then always bring that notebook into the doctor's visit because I'd like you to... I'd like to know the day that you started any medicine, whether it's ARIKAYCE or something else. I'd like to know the day you started having a particular side effect, what was the side effect? How close did the side effects start appearing after you took your dose? Those little details can be very, very helpful to me, the physician, to help manage these side effects, so keeping good records, which it sounds like you did, is very important.

Self-advocating and tracking.

Absolutely. Well, let's learn a little bit more about ARIKAYCE now and more, I mean, we've got some patient support that's really important, and honestly is not available to everyone for every new medication that they start on, so I think these support programs have been so helpful to my patients and I've gotten their feedback, so I know how important it is. So, there's the ARIKAYCE Knowledge Moves program, and if you're looking to learn more about ARIKAYCE, then this program is great for you 'cause it provides information and some educational resources, patient stories like yours, Judy, and other patients, and tips on how to have conversations with your doctor, if you're thinking about going to... Incorporating ARIKAYCE use into your drug regimen and you can always visit ARIKAYCE.com to enroll in this program.

And I think the feedback from my patients has been that that program has been hugely beneficial to them at the beginning of their process and through the process when ARIKAYCE was being utilized, and the next step then is if you and your doctor decide to go ahead and start on ARIKAYCE, then there's another support program called the Arikares support program, and this program is once you have been prescribed are case by your physician, this program kicks in and provides you additional information and support, and you'll be assigned a care team that's totally dedicated to helping you along the journey with ARIKAYCE in your treatment regimen, and they help in a variety of ways, from navigating your insurance coverage to using your nebulizer, which is a little different than most nebulizer equipment that you might have encountered up to this point in time, and you can call 1-833-ARIKARE.

And someone is there joining on the spot to help you in this enrollment process over the phone. And, again, to a patient, I have personally had feedback from them that they felt that this was a very supportive program for them once they started on... Or were getting ready to start on ARIKAYCE. And so, on this slide, if you wanna use your smartphone and scan this code to hear more stories from patients about their journey once they started ARIKAYCE, you can listen to that and watch that after the program is completed at ARIKAYCE.com. So... Judy, we've talked about a lot of different things in this program, but now that we're sort of coming to the end in talking about these support programs that are available, did you have experience with any, or utilize any of these support programs?

Oh, Dr. Huitt, absolutely. I utilized all the resources I could get. And it was very helpful to me. I enrolled in the Arikares Support Program, with the onboarding of ARIKAYCE into my program, they were helpful with providing guidance and financial support for starting off on the right foot with all of this, the actual training with the Arikares trainer was really easy. She made it almost seamless and easy to do to get started, and I was ready to receive the information. I had done my homework. So they're all great resource. Arikares coordinator is constantly checking and making sure that you have all the information you need, that you have the proper tools to work with. If you have any questions, you can go back to them with everything... It works out very well for me. I found support groups online to sort of add additional information to my progress to and to the Arikares and how it worked, and that was also very helpful. Community blogs and community resources are just an added benefit to the whole program. Back to you, Doctor.

Perfect. And you said it all. I think providing our patients with the resources to empower themselves, to advocate for themselves, to see whether this is the right product for them, and if it's deemed that it is the right product for them, to get even more information about how to navigate... Navigate this whole journey, because sometimes it can be daunting, let's not lie, and I think it's so important to be empowered and have information. Information is power, as we say. So let's summarize what we've spent this last several minutes talking about, and... So we'd started this program out talking about Mac lung disease and that it can be a serious condition, and the reason it can be serious is 'cause it can cause significant inflammation in the lungs and cause changes to the lung tissue that may be permanent, and that's what we want to avoid. So, we need to be sure what is the status of your lung infection, and do we need to start on treatment right away, or if it's serious, we shouldn't just sit and do as we say, watchful waiting. We should get busy and figure out what to do and what treatment regimen to start you on.

And some people are at greater risk for getting Mac lung disease due to their underlying conditions such as bronchiectasis and COPD that we alluded to earlier. Some people have a cough, little cough... Comes and goes, it's not really so severe, but generally people with Mac lung disease have cough, have fatigue and no response or minimal response to things that your doctor may try, and so that's when you wanna think, surely, "Gosh, do we need to look as to whether I have Mac lung disease?" and if we do decide you have Mac lung disease, then fortunately, we have a 2020 document called The NTM treatment guidelines that tells your doctor and you what the treatment recommendations are to start a multi-drug treatment regimen, if we're going to start rather than watchful waiting, and once you've started your three drug regimen, if after six months, your sputum cultures have not converted to negative, then the NTM treatment guidelines recommend adding ARIKAYCE.

And it's important to know that this was so important in those of us that treat NTM Mac lung disease, that ARIKAYCE is the first and only FDA-approved treatment designed specifically for people who don't respond to a multi-drug regimen alone for the first six months, if you haven't converted your student to negative by that time, it's time to think about adding ARIKAYCE. Now, ARIKAYCE is associated with risk of increased respiratory adverse infections or reactions, including allergic inflammation of the lungs, coughing up blood, severe breathing problems and worsening COPD. Be sure that you talk to your doctor if you have any questions about ARIKAYCE, including possible side effects that may occur, and you can also visit ARIKAYCE.com for more information and support. So, we've had quite a journey together, Judy, and after hearing all of this, and thank you so much, first of all, for sharing your experiences, 'cause I think they're so helpful to patients... They're helpful to me actually. Do you have any final thoughts? Words of wisdom?

Well, I don't know how full of wisdom I am, Dr. Huitt, but thank you for thinking so. I am a big proponent of self-advocating and making sure that you understand the parameters that you're entering into, that you understand what you're taking, why you're taking, what is... You had mentioned earlier what the goals are for both you and your doctor that they have an understanding also of your own personal investment, and what are you willing to commit to to your own well-being to have a potential for successful outcome? That was my investment, my doctor understood I was in for the fight, and I haven't been around this long with this NTM. If we can shorten people's journey to get to this point, it certainly would be impactful for many, many people, and I know that from my own personal experience. I just think, again, it's really important to keep hope here even when you're down for the count, and I think it's even more important to have perseverance top of mind, to keep after it, to keep asking questions, to keep advocating, to seek out the answers.

If you're not getting the answers from your doctor, ask them another way. Ask them to follow a different track or to seek out the answers. I had one doctor look at me and ask me if I wanted to be his assistant, and I thought, "That's kind of a compliment. I don't if I'm irritating him or not, but if we can get to the end goal, that's fine with me." And I just think it's really important, as you mentioned, to track it, to keep a log book specific, you might think the small detail doesn't matter, but it really does. And I think having good people around you to help you get to that end goal is very important, too. Arikares and the whole format and the way that it's put together has been very, very helpful to me personally, Dr. Huitt. I really appreciate... I learned something every day. Thank you.

Well, great. And, all my patients always educate me, and it's so important to the physician community to constantly be challenged, honestly, and educated by our patients as well, because honestly, we can always learn right along with you, and we should always be learning, we should strive for new information. So, I think that's the conclusion of the formal presentation, so I'm going to toss back to our moderator, Nicole.

Yes, and thank you to you both for sharing this important information with all of us this evening, as well as your perspectives. I know this is invaluable to all of us. So, once again, thank you to both. So, this concludes our presentation this evening, thank you again so much to our special guests, and for all of you who joined us today. We have additional programs coming up in the future, so we hope to see you again. Thank you.

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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.