ARIKAYCE targets the MAC infection directly in your lungs

  • ARIKAYCE is an inhaled antibiotic taken with a nebulizer

  • ARIKAYCE is different because the antibiotic is contained inside tiny particles called liposomes which help it reach the lungs

  • When inside the lungs, the liposomes enter the body’s cells (called macrophages) and release the antibiotic to fight the MAC bacteria where it lives

Get the whole story: Watch ARIKAYCE in action

  • Lynn

    “ARIKAYCE just felt like a hopeful tool. Maybe this would work for me.”

    Judy, a real ARIKAYCE patient

EXPLORING ARIKAYCE

Behind the liposome

Join Judy, a real patient on a personal journey to understand the science behind ARIKAYCE, as she speaks with a medical researcher about the development of this therapy.

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.

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Next: ARIKAYCE study results
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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.