ARIKAYCE is proven to help people test MAC-negative and stay MAC-negative, even after completing treatment

Studied in people whose initial multidrug treatment wasn’t successful

All of the people in the ARIKAYCE study had been on treatment for MAC lung disease for at least 6 months, but were still testing positive for MAC.

224 people then added ARIKAYCE to their multidrug treatment and 112 continued to take a multidrug treatment alone.

  • Study objectives: The study looked at the number of people in each group who tested MAC-negative over time. The study also looked at how long people stayed MAC-negative after completing treatment
  • Additional objectives: Improvement in the distance people walked after 6 minutes and in overall health, daily life, and perceived well-being in people with lung disease (as measured by a questionnaire)*
*6-minute walk test and St George’s Respiratory Questionnaire.

In a clinical study, adding ARIKAYCE helped more people test MAC-negative for 12 months and beyond

PRIMARY RESULTS

ADDITIONAL RESULTS

29%
(65/224)
8.9%
(10/112)
18.3%
(41/224)
2.7%
(3/112)
16.1%
(36/224)
0%
(0/112)

6 MONTHS

12 MONTHS

at treatment completion

3 MONTHS

after completing treatment

3X

Adding ARIKAYCE helped 3x as many people test MAC-negative

7X

Adding ARIKAYCE helped 7x as many people test MAC-negative

ONLY

Only people who added ARIKAYCE tested MAC-negative

Percentage comparison.

Additional study results:

In the ARIKAYCE clinical study, there was no improvement in the 6-minute walk test and St George’s Respiratory Questionnaire measurements at the end of 6 months.


ARIKAYCE EXPLORED

Is ARIKAYCE for you? A medical expert explains the data

Real patient Judy goes on a fact-finding mission and speaks with a MAC lung disease specialist who treats patients with ARIKAYCE. Learn how the results from a clinical study inform this doctor’s decision to prescribe ARIKAYCE.

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.

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.

  • See the findings from a side effects management survey
  • Get help talking about ARIKAYCE with your doctor. Use the Doctor Discussion Guide
Next: The ARIKAYCE treatment plan
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.