Mentorship | AMVUTTRA® (vutrisiran)

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Cardiomyopathy (ATTR-CM) Polyneuropathy (hATTR-PN) Cancel

ATTR amyloidosis, or ATTR, is a progressive, debilitating, and fatal disease which occurs when a protein called transthyretin, or TTR, misfolds and builds up in the body. There are two types of ATTR amyloidosis; hereditary and wild‑type. Hereditary ATTR amyloidosis is caused by an inherited gene variant that runs in families, whereas with wild‑type ATTR amyloidosis, the cause is unknown, but it is believed to develop with age.

To understand ATTR amyloidosis it's important to know how transthyretin, or TTR, normally works in the body. TTR is a protein that is made primarily in the liver. The liver produces TTR proteins, which are released into the bloodstream. Under normal conditions, TTR helps carry vitamin A and other substances throughout the body. A healthy TTR protein is made up of four parts that fit together to form the functional TTR protein.

In ATTR amyloidosis, TTR proteins become unstable. Instead of holding their shape, they break apart and misfold. These misfolded TTR pieces can stick together and form amyloid fibrils. Over time, these amyloid fibrils can build up as amyloid deposits in different parts of the body including the heart, nerves, digestive system, and other organs, leading to a range of debilitating symptoms.

AMVUTTRA is a prescription medicine that treats the cardiomyopathy of wild‑type or hereditary ATTR and the polyneuropathy of hereditary ATTR in adults.

AMVUTTRA is a silencer that works in the liver, where most TTR protein is made, to reduce its production at the source. It uses advanced technology to selectively target and enter the liver, reducing the amount of TTR produced and circulated in the blood.

Less TTR protein in the blood means there is less that can misfold and build up as amyloid deposits in the body. By reducing TTR production at its source, AMVUTTRA helps lower circulating TTR and targets the underlying cause of the disease.

Important Safety Information.

What are the most important things I should know about AMVUTTRA, vutrisiran?

AMVUTTRA can cause low vitamin A levels. Treatment with AMVUTTRA lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day.

You should not take more than the amount of vitamin A recommended by your doctor. Low vitamin A levels can affect vision. If you have problems with your vision, for example night blindness, while taking AMVUTTRA, talk to your doctor. Your doctor may refer you to an eye specialist.

What are the common side effects of AMVUTTRA?
The most common side effects of AMVUTTRA were pain in the arms or legs, pain in the joints, shortness of breath, and low vitamin A levels. These are not all the possible side effects of AMVUTTRA. Talk to your doctor about side effects that you experience. You are encouraged to report negative side effects of prescription drugs to the U.S. Food and Drug Administration, FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Indications.

What is AMVUTTRA?
AMVUTTRA is a prescription medicine that treats the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis, ATTR-CM, in adults to reduce heart-related death, hospital stays and urgent visits. Polyneuropathy caused by hereditary transthyretin-mediated amyloidosis, hATTR-PN, in adults.

For additional information about AMVUTTRA, please see the full prescribing information at www.amvuttra.com

With ATTR-CM, there’s no time like the present to consider your future. How will you choose to start your journey.

AMVUTTRA treats the cardiomyopathy of ATTR amyloidosis in adults.

It was proven to help people with ATTR-CM live longer and have fewer heart-related hospital visits over 3 years, compared to placebo.

AMVUTTRA is the first and only silencer for ATTR-CM.

It reduces TTR at the source and requires just 4 injections a year.

So you can focus on the journey ahead.

AMVUTTRA lowers the amount of vitamin A in your blood which can affect vision. Other side effects include pain in the arms or legs, pain in the joints, and shortness of breath.

Now’s the time to think about your future with ATTR-CM. Take the next step and talk to your doctor about AMVUTTRA today. AMVUTTRA: just the ticket for ATTR-CM.

Most patients pay $0 out of pocket. Learn more at AMVUTTRA.com/ticket.

Art:
I've known about this disease for 50 years because my grandfather and my father both died from it.

Tim:
When it first dawned on me that something was odd, I was walking on level ground and having to stop and hold on to a telephone pole to catch my breath. It didn't make any sense.

Art:
In the back of my mind I was hoping I didn't have ATTR, but one day I had a shortness of breath episode where it dropped me to my knee and I thought I was having a heart attack. I said to the cardiologist, so tell me, doc, do I have it? And he said, yes, you have it. It was a real jolt to my system and uh it was very difficult to hear.

Tim:
The first question I had for my doctor was, “What is this going to do to my lifespan?”

Art:
I didn't know how long I had to live, and I was not going to waste a day, so I just kept doing more and more research.

Tim:
I remember the cardiologist saying, “There's some drugs out on the market. We're very optimistic about them.”

Art:
You have to trust your doctor. You have to have full confidence in your doctor. You have to have a team, and you have to trust in your team.

Tim:
Finding the right doctor is paramount. I follow their instructions. They answer my questions. It's a good relationship.

Art:
You have to be your own advocate. If you have a concern or a question and somebody doesn't answer, don't stop. Keep going. Find somebody that will give you the answers that you need.

Tim:
It’s vital to have a strong medical team. That’s one thing that you can take out of the equation. You don’t have to sweat that. You can focus on other things. Staying active. Getting exercise. Eating well.

Art:
I'm probably not a real good patient because I do too much research, so I go in with what I think is a lot of knowledge, but my doctor’s really receptive. He understands that I'm just trying to live my life, and he's trying to make that happen.

Announcer:
Important Safety Information and Indications.
Important Safety Information.
What are the most important things I should know about AMVUTTRA® (vutrisiran)?

AMVUTTRA can cause low vitamin A levels.
Treatment with AMVUTTRA lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day. You should not take more than the amount of vitamin A recommended by your doctor.
Low vitamin A levels can aﬀect vision. If you have problems with your vision, for example, night blindness while taking AMVUTTRA, talk to your doctor. Your doctor may refer you to an eye specialist.

What are the common side eﬀects of AMVUTTRA?
The most common side eﬀects of AMVUTTRA were pain in the arms or legs, pain in the joints, shortness of breath, and low vitamin A levels.
These are not all the possible side eﬀects of AMVUTTRA. Talk to your doctor about side eﬀects that you experience. You are encouraged to report negative side eﬀects of prescription drugs to the US Food and Drug Administration (FDA). Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Indications.
What is AMVUTTRA?
AMVUTTRA is a prescription medicine that treats the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce heart related death, hospital stays and urgent visits.
Polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
For additional information about AMVUTTRA, please see the full Prescribing Information at amvuttra.com.

Announcer:

A guide to receiving AMVUTTRA

Please view the entire video for Important Safety Information and visit amvuttra.com for the full Prescribing Information, or scan the QR code at the end of the video.

Patient 1:

If you’re getting ready to receive AMVUTTRA, you want to feel prepared. I’m here to walk you through some basic information.

AMVUTTRA is a prescription medicine that is given as an injection once every 3 months by a healthcare provider.

That’s only 4 times a year. You and your doctor will decide where is most convenient to receive your treatment.

I visit my doctor’s office, but some people go to a nearby clinic. It can vary based on your doctor or insurance. If your insurance covers it, you may even be able to receive AMVUTTRA at home from a mobile healthcare provider.

If you’re like me, you may worry about getting an injection. Here’s what to expect.

AMVUTTRA comes as a prefilled syringe. It’s given by a healthcare provider so you can feel confident you’re getting the full dose.

AMVUTTRA is given as an injection with a short needle, in the upper arm, thigh, or abdomen. I usually get mine in my arm.

After cleaning the area, the healthcare provider will pinch your skin. Next, they’ll give you AMVUTTRA subcutaneously, which means just under the skin.

The administration process will only take a few minutes. It’s a good idea to set up your next appointment before you leave.

If you’re receiving AMVUTTRA at home, there are a few more things you need to know.

Patient 2:

Let’s talk about at-home administration by a healthcare provider. My insurance covers it, but it’s not an option for everyone.

If you’re eligible to receive AMVUTTRA at home, the specialty pharmacy will call to set up your appointment. Now, before your appointment, the specialty pharmacy will deliver AMVUTTRA. You may need to sign for the package, so make sure someone is home.

Your first shipment of AMVUTTRA will include a welcome packet and supplies for your appointment. Don’t open the medicine carton or syringe.

AMVUTTRA can be stored in the fridge or at room temperature. If you keep yours in the fridge like me, make sure you have a dedicated space for it. And don’t freeze it.

On the day of your appointment, take AMVUTTRA out of the fridge 30 minutes before the nurse arrives. That way it can reach room temperature. Then prepare a space to receive your injection.

You’ll need a clean surface and a comfortable seat. Now, once the nurse shows up, they’ll clean the injection site, pinch your skin, and use the short needle to give you AMVUTTRA subcutaneously. That means just under the skin.

The administration process will only take a few minutes. And before the nurse leaves, it’s a good idea to confirm your next appointment.

Announcer:

AMVUTTRA may cause reactions at the injection site such as bruising, redness, pain, itching, and warmth.

Questions about accessing treatment? Contact Alnylam Assist. In case of an emergency, call 9-1-1.

Important Safety Information and Indications.

Important Safety Information.

What are the most important things I should know about AMVUTTRA® (vutrisiran)?

AMVUTTRA can cause low vitamin A levels.

Treatment with AMVUTTRA lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day. You should not take more than the amount of vitamin A recommended by your doctor.

Low vitamin A levels can aﬀect vision. If you have problems with your vision, for example, night blindness while taking AMVUTTRA, talk to your doctor. Your doctor may refer you to an eye specialist.

What are the common side eﬀects of AMVUTTRA?

The most common side eﬀects of AMVUTTRA were pain in the arms or legs, pain in the joints, shortness of breath, and low vitamin A levels.

These are not all the possible side eﬀects of AMVUTTRA. Talk to your doctor about side eﬀects that you experience. You are encouraged to report negative side eﬀects of prescription drugs to the US Food and Drug Administration (FDA). Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Indications.

What is AMVUTTRA?
AMVUTTRA is a prescription medicine that treats the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce heart related death, hospital stays and urgent visits.
Polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

For additional information about AMVUTTRA, please see the full Prescribing Information or scan the QR code.

Learn more about treatment with AMVUTTRA at amvuttra.com.

Tim:
I go to a medical facility every three months to get my injection.

Ed:
Once every three months works, doesn't it?

Penny:
Yeah. We can plan around it.

Ed:
It gives us freedom to do what we want to do.

Kelly:
I know when I'm getting these injections, I can schedule around it, not pause my life for the treatment that I need.

Ed:
We try to make a day of it. Either lunch or visiting family or friends.

Tim:
The guesswork is taken out of it. They call me. “Hey, dude. It's time. Time to come back in.”

Art:
It's on my phone. It’s on my calendar. It’s not something I forget. In all the years, I've never missed or forgot an appointment.

Scott:
My ritual, every single time is I get in the car. I put on the music very, very loud. I sit there for a minute after I'm parked, and then I walk in.

Kelly:
The nurse comes in and asks me questions about my health and how I've been feeling.

Tim:
They're on their game, they know what they're doing. You come in and you sit down.

Scott:
And then they ask where I would like it. They always give me the option of my abdomen, my thigh, or the back of my arm.

Tim:
It’s subcutaneous, and that’s ok with me.

Ed:
I scheduled my next appointment three months later, and we're off and running.

Kelly:
Honestly, it takes an hour out of my day and that includes the time to get there, parking and everything else.

Tim:
I really appreciate that the injection is given by a health care provider.

Kelly:
That way I know I'm getting the proper dose.

Penny:
And it’s getting put in the correct area. I know that's important.

Art:
My doctor told me the most common side effects are pain in the arms and legs, pain in the joints, shortness of breath, and low vitamin A levels.

Kelly:
So my doctor explained to me that those low vitamin A levels can affect your vision. So I take a vitamin A supplement every day.

Scott:
It might be one of the only times that after I got an injection, I walked down the hall, and I called my wife and I said, “I can't tell you how happy I am.” Happy to be given something that I truly believed would make a difference.

Announcer:
Important Safety Information and Indications.

Important Safety Information.

What are the most important things I should know about AMVUTTRA® (vutrisiran)?

AMVUTTRA can cause low vitamin A levels. Treatment with AMVUTTRA lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day.

You should not take more than the amount of vitamin A recommended by your doctor. Low vitamin A levels can aﬀect vision. If you have problems with your vision, for example, night blindness while taking AMVUTTRA, talk to your doctor. Your doctor may refer you to an eye specialist.

What are the common side eﬀects of AMVUTTRA?

The most common side eﬀects of AMVUTTRA were pain in the arms or legs, pain in the joints, shortness of breath, and low vitamin A levels. These are not all the possible side eﬀects of AMVUTTRA. Talk to your doctor about side eﬀects that you experience. You are encouraged to report negative side eﬀects of prescription drugs to the US Food and Drug Administration (FDA). Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Indications.

What is AMVUTTRA?

AMVUTTRA is a prescription medicine that treats the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce heart related death, hospital stays and urgent visits.
Polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

For additional information about AMVUTTRA, please see the full Prescribing Information at amvuttra.com.

Scott:
It took me easily a month to really understand the severity of the disease.

Tim:
There is no getting around the diagnosis. I’m gonna hold on to what I’m doing as much as I can for as long as I can.

Scott:
My doctor said your ATTR is going to be a journey for the rest of your life. The good news is, for the first time in a very long time, I can look you in the eyes and tell you there's no cure, but we have treatment options.

Tim:
I first heard about AMVUTTRA doing my own research. My cardiologist told me that our goal was to slow the progression of the disease.

Scott:
I was all in.

Tim:
I was completely on board.

Scott:
When I got that first dose, I felt relief. I knew that by getting AMVUTTRA, I was doing something proactive to fight my disease.

Tim:
My doctor explained that the most common side effects are pain in the arms or legs. Pain in the joints. Shortness of breath and low vitamin A levels.

Scott:
Because low vitamin A levels can affect vision, my doctor also has me take a daily vitamin A supplement.

Tim:
I have a social circle of buddies. We hike. We get together. It's definitely slower with more rest stops. I still experience shortness of breath. Stamina continues to be an issue, but we're still out there.

Scott:
When I was diagnosed, I really thought I was going to have to stop walking. But I can walk. And I love walking. I listen to really loud rock music. That's what makes me happy. So being able to continue that was very important to me. I know ATTR is progressive. Whenever I have a new symptom or a symptom gets worse, I talk to my doctor.

Tim:
I've taken all the steps I can to put the odds in my favor. My ten-year-old grandson doesn't live near me, and I want him to know me, and I want to know him. I'm quietly confident that this is the right path to be on, to watch my grandson grow up.

Scott:
I have this disease, okay. I don't feel bad about it. I just keep going. I'm unbelievably grateful that I could actually go on walks. I can go out to dinner with my wife. I can plan things. And being on AMVUTTRA enables me to be able to do that.

Announcer:
Important Safety Information and Indications.

Important Safety Information.

What are the most important things I should know about AMVUTTRA® (vutrisiran)?

AMVUTTRA can cause low vitamin A levels. Treatment with AMVUTTRA lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day.

You should not take more than the amount of vitamin A recommended by your doctor. Low vitamin A levels can affect vision. If you have problems with your vision, for example, night blindness while taking AMVUTTRA, talk to your doctor. Your doctor may refer you to an eye specialist.

What are the common side effects of AMVUTTRA?

The most common side effects of AMVUTTRA were pain in the arms or legs, pain in the joints, shortness of breath, and low vitamin A levels. These are not all the possible side effects of AMVUTTRA. Talk to your doctor about side effects that you experience. You are encouraged to report negative side effects of prescription drugs to the US Food and Drug Administration (FDA). Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Indications.

What is AMVUTTRA?

AMVUTTRA is a prescription medicine that treats the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce heart related death, hospital stays and urgent visits.
Polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

For additional information about AMVUTTRA, please see the full Prescribing Information at amvuttra.com.

PETE: I love the game of football. I love the competition. I love the physicality of it, and I love it for the opportunities it offered me. It changed my life.

PETE: On the football field, I was known for being a hitter, a big hitter. It's not the size of the dog in the fight, you know? It's the size of the fight in the dog. And I was that dog.

PETE: Football got me out the hood. It got me a scholarship to college, got my kids through college. So football's a family affair.

KARI: I fell in love with Pete because of his heart.

KARI: Pete has ATTR cardiac amyloidosis. Makes the heart unable to function normally. That's been tough to see…a husband that was so strong. Now be the guy that has struggled…

PETE: The pain…Um, riddled my body for years, but I thought it was football. I thought it was, you know, being a hard hitter and playing on artificial turf.

KARI: Being out of breath, just coming up the stairs, like, what is going on? The cardiologist said, I want to talk to you about your heart.

DR MOHAN: How are things going? How have you been? ATTR cardiac amyloidosis is an abnormality of the heart muscle that's due to protein buildup.

DR MOHAN: When a protein that we all have called TTR, transthyretin, becomes abnormal and separates into little pieces that form fibrils, which are essentially toxic to the heart muscle cells. ATTR cardiac amyloidosis can be hereditary. There is the non-hereditary type, which is much more common, and then the hereditary type, which we see in people who have a gene mutation. ATTR is easy to miss because many times it affects other parts of the body before it gets to the heart. Things like musculoskeletal issues, carpal tunnel, spinal stenosis.

KARI: He would do check marks and just, “I have that. I had carpal tunnel. I have trigger finger.” One day he called me from his office, and he couldn't even get a sentence out. And I'm like, is he having a stroke or is he having a heart attack? It's very scary.

PETE: I went in for the worst news. You know, I may have to have a heart transplant. He took some tests…

PETE: …and he told me. We're going to put a hold on that for now.

PETE: And you talk about exhaling. It's, uh…There weren't any words.

DR MOHAN: After we made the diagnosis of ATTR cardiac amyloidosis, we started Pete on a drug called AMVUTTRA. AMVUTTRA is a TTR silencer, and it reduces TTR production in the liver. There is no cure for ATTR cardiac amyloidosis, but AMVUTTRA does slow the disease progression down.

KARI: As a caregiver. It's tough. At times. You're like, so today looks different than yesterday. Is this what tomorrow's going to look like? But we're doing this journey together.

PETE: I want people to be aware of this disease. About 4% of black Americans carried the V122I gene. It is hereditary, so it could be in my family. I could pass it on to my children. My son had the hereditary gene variant. He didn't know and I didn't know.

PETE: I wanted to tell my story so that others have hope….

PETE: To hear…that there were treatment options meant that I had a fighting chance. AMVUTTRA is my treatment for ATTR cardiac amyloidosis. I go to a medical center every three months to get my injection given by my health care provider. They ask where I want it. My abdomen, thigh or upper arm. The most common side effects are pain in the arms and legs, pain in the joints, shortness of breath, and low vitamin A levels. Because low vitamin A levels can affect vision, my doctor has me take a daily vitamin A supplement as well.

PETE: AMVUTTRA slows the disease…it gives me time.

PETE: Hi there!

KARI: Hi Zoe!

PETE: This is the reason…

PETE: …I'm trying to stay around. Oh, Golly.

KARI: He has his energy. Not 100%. Because we know that this disease is progressive, but each day is a gift.

PETE: Some days are better than others, but I'm hopeful. There is hope for a longer life.

PETE: When I think about my legacy…

PETE:…I want to be that guy that my grandkids are proud of. That heart that I played with, that I love my kids with that Rosalee and Woody Shaw gave me, that's very, very important to me. I'm a fighter and I'm fighting this thing now with AMVUTTRA.

Important Safety Information and Indications

Important Safety Information.

What are the most important things I should know about AMVUTTRA® (vutrisiran)?

AMVUTTRA can cause low vitamin A levels. Treatment with AMVUTTRA lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day.

You should not take more than the amount of vitamin A recommended by your doctor. Low vitamin A levels can affect vision. If you have problems with your vision, for example, night blindness while taking AMVUTTRA, talk to your doctor. Your doctor may refer you to an eye specialist.

What are the common side effects of AMVUTTRA?

The most common side effects of AMVUTTRA were pain in the arms or legs, pain in the joints, shortness of breath, and low vitamin A levels. These are not all the possible side effects of AMVUTTRA. Talk to your doctor about side effects that you experience. You are encouraged to report negative side effects of prescription drugs to the US Food and Drug Administration (FDA). Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Indications.

What is AMVUTTRA?

AMVUTTRA is a prescription medicine that treats the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce heart related death, hospital stays and urgent visits.
Polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

For additional information about AMVUTTRA, please see the full Prescribing Information at amvuttra.com.

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