Results in adults and children with Type 2 or 3 SMA

Results in adults and children with Type 2 or 3 SMA

Results in adults and children with Type 2 or 3 SMA

Children and adults patients on Evrysdi

Adults and children improved their motor skills and upper limb function when taking Evrysdi, an at-home treatment

SUNFISH is a 2-part, placebo-controlled study in people with Type 2 or 3 spinal muscular atrophy (SMA). The trial included people 2 to 25 years: the first placebo-controlled trial in adults and children older than 9 years of age with SMA.

Learn more about the trial

Evrysdi improved or preserved motor skills

EVRYSDI IN ADULTS AND CHILDREN (SUNFISH PART 2)

Change in motor function score over 12 months vs placebo
As measured by MFM-32

Change in motor function score over 12 months chart

Adults and children taking Evrysdi showed improved motor function compared with placebo

Adults and children taking Evrysdi showed improved motor function compared with placebo

*In some studies, including this one, if someone’s data cannot be collected on time for any reason, that person’s progress cannot be counted in that part of the study. This chart includes only the information that was collected on time.
^†Adults and children not taking Evrysdi took a placebo, a substance that has no active medication and is often used in studies.
^‡This 95% CI (confidence interval) means that we are 95% confident that the actual average change in MFM-32 with Evrysdi will be between 0.30 and 2.81 points higher than with placebo.

MFM-32 stands for Motor Function Measure-32 Items.

The Motor Function Measure-32 Items (MFM-32) scale measures motor function abilities that relate to important daily functions. It evaluates 32 different elements to assess motor movement across 3 categories:

Standing/transfer movements

upper and lower body movements image

Upper/lower body movements

hand and foot movements image

Hand/foot movements

The scale is designed to capture change for a broad range of people, including those who can and cannot walk.

SUNFISH PART 2

Change in motor function score over 24 months
As measured by MFM-32

Change in motor function score over 24 months chart

1.83-point average change in MFM-32 score from the start of the study with Evrysdi.

This information is considered exploratory. This means it was not designed to show a treatment effect so conclusions cannot be drawn.

*In some studies, including this one, if someone’s data cannot be collected on time for any reason, that person’s progress cannot be counted in that part of the study. This chart includes only the information that was collected on time.
^†Adults and children not taking Evrysdi took a placebo, a substance that has no active medication and is often used in studies. People in this group received placebo for 12 months followed by Evrysdi for 12 months. The period of time on Evrysdi is not included in this chart. The follow-up period was not placebo controlled.

MFM-32 stands for Motor Function Measure-32 Items.
NA stands for not applicable.

Shaniqua, lawyer, thrill seeker, and warrior for women with disabilities, living with Type 3 SMA

“Since starting Evrysdi, I have noticed changes in my motor function. I can better push up on my arms and stand when transferring out of my wheelchair.”

Shaniqua, lawyer, trusted friend, and warrior for people with disabilities, living with Type 3 SMA

Evrysdi improved upper limb strength important for activities of everyday life

EVRYSDI IN ADULTS AND CHILDREN (SUNFISH PART 2)

Change in upper limb function score over 12 months vs placebo
As measured by RULM

Change in RULM function score over 12 months chart

Adults and children taking Evrysdi showed greater improvement in upper limb function compared with placebo

Adults and children taking Evrysdi showed greater improvement in upper limb function compared with placebo

*In some studies, including this one, if someone’s data cannot be collected on time for any reason, that person’s progress cannot be counted in that part of the study. This chart includes only the information that was collected on time.
^†Adults and children not taking Evrysdi took a placebo, a substance that has no active medication and is often used in studies.
^‡This 95% CI (confidence interval) means that we are 95% confident that the actual average change in RULM with Evrysdi will be between 0.55 and 2.62 points higher than with placebo.

RULM stands for Revised Upper Limb Module assessment.

The Revised Upper Limb Module (RULM) evaluates strength in arm movements and the ability to perform specific tasks. It was specifically designed for people 2.5 years and older living with SMA who have varying levels of muscle weakness and includes tests such as:

pick up objects image

Picking up tokens
Placing a coin or other small object into a cup
Pushing a button

using a pencil image

Using a pencil
Tearing paper
Reaching to the side

raise cup to mouth image

Raising a cup to mouth
Bringing hands from lap to table
Bringing a hand to shoulder
Bringing a hand above shoulder
Bringing both arms above head

open plastic container image

Opening a container
Lifting and moving 1/2-lb and 1-lb weights*
Bringing 1-lb weight from lap to table or eye level*
Bringing 1-lb and 2-lb weights above shoulder*

*Weight is approximate and has been converted from grams to pounds.

SUNFISH PART 2

Change in upper limb function score over 24 months
As measured by RULM

Change in RULM function score over 24 months chart

2.79-point average change in RULM score from the start of the study with Evrysdi.

This information is considered exploratory. This means it was not designed to show a treatment effect so conclusions cannot be drawn.

*In some studies, including this one, if someone’s data cannot be collected on time for any reason, that person’s progress cannot be counted in that part of the study. This chart includes only the information that was collected on time.
^†Adults and children not taking Evrysdi took a placebo, a substance that has no active medication and is often used in studies. People in this group received placebo for 12 months followed by Evrysdi for 12 months. The period of time on Evrysdi is not included in this chart. The follow-up period was not placebo controlled.

RULM stands for Revised Upper Limb Module assessment.
NA stands for not applicable.

My goal was to maintain the strength I had and hopefully slow the progression. Since Evrysdi, I have had improvements in my physical assessments and I’m better able to hold my pen and write more consistently.”

Zack, home chef, music lover, and brewmaster, living with Type 3 SMA

Zack, living with type 3 SMA

Jewelfish trial icon

Learn more about JEWELFISH, an open-label safety study including adults, children, and infants taking Evrysdi who have previously received treatment with other approved or investigational SMA medications.

Trial information

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Inspiring the next generation

As a teacher, Angela motivates children every day. Continuing to be there for her students, her husband, and her friends was one of the many reasons she was motivated to choose Evrysdi.

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Hi, I'm Angela, and I live here in Houston, Texas, and I'm married to the most wonderful man, Justin.

I've always been a person who just has a really independent spirit. And whether it means I'm going to be able to do something myself or if I'm gonna help to put the right people in place to help me accomplish that task, I'm just gonna figure it out.

My doctor and I talked about the goals that I have for my life, and if Evrysdi would help me to potentially achieve those goals. And we talked about kind of the potential side effects that could take place, but also the things that could possibly happen after taking it. After that discussion, we made the choice that I would be taking Evrysdi.

When I think of taking Evrysdi, it fits so perfectly with my "I can" attitude. I like to focus on the things that I can do, and so I can take Evrysdi every day. When I started with Evrysdi, I definitely wanted just to maintain where my current motor function is. I thought about how important my arm and hand function were to my daily living. It allowed me to brush my teeth and put on my own makeup. That's really important to me, because it's less caregiver time. I didn't want to get to a point where I needed to bring in more help or where my husband needed to assist me, 'cause we've built a life around the things that I'm able to do, and I wanted to maintain as much motor function as I can so that I could keep doing the things that are important to me.

I've always been able to feed myself. I got into makeup when I was pretty little, so starting in about sixth grade I had my blue eyeshadow that I was able to do myself and can continue to do that as well, now that I'm in my forties.

I'm really happy with my results on Evrysdi. Every time I go to the neurologist, I do a motor function test, and the last time I went, I actually went up a point, which I don't know if that means anything to you, but I was really excited. I was able to tear a piece of paper, and it was the best sound I'd ever heard, that I was able to do that. I was still able to move the jar of coins from one circle to the other, and I picked up the bag of bird seed, so it was exciting. Over the years, trips to neurologists can kind of be disheartening when you have SMA, because things aren't necessarily getting better. And so to score a point higher on a test and do a little bit better, it means everything.

One thing that's really important to me in this world and my purpose is to kind of eliminate those stereotypes of people with disabilities. And so part of that has always been that I want the illusion of independence. I want, when people look at me, I want them to think, "Man, she does it all." "She's able to accomplish so much." And behind the scenes, there's people that help me do that.

Being a teacher is one of the greatest gifts of my life. I wanna know that I am doing everything I can to maintain motor function so that I can be there for my family, and my students, and my husband. I would tell anyone with SMA to go and talk to their neurologist and talk to them about Evrysdi, because for me, it has been such a good option.

This is Evrysdi in action.

Voicing creativity

Hear a voice-over artist share why he’ll never take Evrysdi® (risdiplam) for granted while living with SMA. Please read Important Safety Information including the Full Prescribing Information for more details.

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I will never take Evrysdi for granted because I know what it was like 40 years ago. And so, having the ability to take that treatment is never wasted on me. I love my work as a voice-over artist. But I'm uniquely qualified for it, I understand technical aspect of it. Having worked at radio stations, I know what I need to have, equipment-wise, room-wise, acoustic-wise. I have friends who are or have been retired, and I have no desire to do that. For me, retirement means doing something that I enjoy, and I've always had work that I enjoy. A little bit technical, a little bit creative, a little bit fun, and this is something that I can do.

I've always done physical work, I've always been active. The last few years of high school, it was, became prevalent and obvious something was wrong. And I decided that, well, I can fix this, I can just go to the gym. So I did. I went to the gym. And on about the second machine, the owner of the gym looks at me and says, "What's your problem?" And I’ll admit, that's really the first time that it hit me. You know what, something's wrong. It was a couple of weeks later with his help I had an appointment at the local neurology center at the university hospital down the road, and that's when it all started. There comes the acceptance, there comes the realization that you need to do all you can do, but it's still never going to go away.

But imagine there's a hundred things that you do every day that are important. You get up in the morning, you stand up, you walk to the bathroom, you kiss your wife, you answer the phone, you hug your kids. There's more than a hundred things, but imagine there's a hundred things that you do. And with this disease, those go away. I found out about Evrysdi because I kept looking for what's coming next. What drove me to try Evrysdi were the good reports I was hearing from the other clinical trials.

When Evrysdi became available, it was clear to me, not only was it going to be the drug for me, but the method of delivery was something that I could do, I could do from home. We've noticed small improvements and I will continue taking it. I've been happy with them, and the doctors have been happy too. If there's one word that I could use to describe the future with Evrysdi, it would be hope.

Embracing the future

For Dan and Milly, their next steps together are exciting. Hear the reason Dan finally chose to treat his SMA and his plans for the bright days ahead..

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It wasn't until I became an adult, that more SMA played a part in my life.

I was originally Dan's caretaker, and it turned into more. He became like my best friend, and from there, I fell in love with my best friend. And that's where we are today.

During the beginning of our relationship, I noticed that he wasn't taking care of his self. So it made me nervous. And I finally stepped in and had to let him know how I felt. And I had a very emotional conversation with him, just letting him know I love him. I wanna spend the rest of my life with him.

What really motivated me to start taking treatment was Milly actually telling me how important it was for me to be healthy for her. And so I did seek treatment, and meeting with my doctor and my neurologist. Since taking Evrysdi, I definitely have noticed my improvements, and my doctors have been very happy with the progress as well.

What we really like about Evrysdi is, it fits into our daily life. It's a liquid I take every day. Even if we travel, we just take it with us.

We give him his oral medication every morning.

Every morning after breakfast.

I work for the largest entertainment company in the world. I work at home, so it’s a long commute to my office in the house. And I pretty much work all day, take a break for lunch. And as soon as I'm done with work, I just wanna be with my wife.

Since we're so spontaneous and he likes to do things randomly, we go to the park on Saturdays, sometimes the beaches, on Wednesdays and Thursdays. Go to the dog park, just a random day. We love to play games, especially cards, because we're really competitive. We always say goodnight and I love you. We always say I love you.

Always.

Milly told me about Evrysdi. She is studying to be a doctor. So, she understood all the molecular – all the biological stuff and how it works.

I think my biggest encouragement to other adults with SMA would be to seek out Evrysdi. Talk to your doctor, because even if it doesn't have the same effects physically as it does for me, it definitely will give you hope. And that's the biggest thing for me anyways.

I am grateful for everything that I have. I want to keep moving toward my future with my family.

Pursuing justice for others

Listen to Shaniqua, a disability rights attorney and Evrysdi® (risdiplam) patient, discuss living with SMA.Please read Important Safety Information including the Full Prescribing Information for more details.

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My name is Shaniqua. I am attorney living in Silver Spring, Maryland, and I have spinal muscular atrophy Type 3, also known as SMA.

When I think about Evrysdi, I think about hope. You know, hope to maintain strength, hope for the SMA community.

I was diagnosed with SMA around the age of two and a half. I always knew that I would lose motor function but I didn't know what it would look like. In middle school I still could walk longer distances. I noticed over the years that I was slowly losing some of my motor functions. So I use my wheelchair normally when I'm working, I'm at my desk, but sometimes I do get up and I walk around. I walk to the sink to start cooking, so I really like to cook. And so I can walk to my bedroom to get in the bed and walk to the restroom.

Taking Evrysdi treatment is important to me because it's me considering my health and how important my health is and me doing something about it, me taking the action, and that hopefully I'll be able to maintain my strength. I spoke to my doctor about Evrysdi. Ultimately, the decision was mine whether to take the treatment or not, and I just ultimately decided that it was the best thing for me and that I was very hopeful that it would help.

My goal is to continue and maintain some of the things that I'm able to do on my own. I have always loved to cook. I love to make meals for myself and sometimes I like to make meals for my family. It's just a joy seeing their faces after I've cooked something, so I really like being able to do that on my own. I have always been able to brush my teeth, wash my face, do my hair.

The physical therapist normally tests my motor skills twice a year. Since starting Evrysdi, I have seen improvements in my motor functions. I've noticed that I can pick up small objects and open containers, and also I've seen that it's easier for me to get up out of my chair to a standing position. I'm happy with my experience on Evrysdi. It works for my life because it's an oral medication and I can just take it at home and I don't have to go out or to a doctor or to a hospital to take the drugs. I'm not confined to taking my medication at one place.

When I think about Evrysdi, I think about being able to have hope, and every time I take the medication, I'm hoping that the strengths that I do have will be maintained.

This is Evrysdi in action.

Owning the day

For the Bonner family, spending time together is their biggest priority. Hear why Evrysdi is the right choice for their life on the go.

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My name is Erin and this is my husband, Kevin. We live on Long Island with our two sons, Bear, who is seven and has Type 2 SMA, and Sam, who is one and a half, unaffected.

My name is Bear. I have SMA Type 2.

I think that Bear is a joyful person because he's able to find humor in almost anything, and he loves creating his own jokes, and he loves making other people laugh.

He always wants to go to a museum or an aquarium. He's always curious, always wants to see those things and do those things.

I always love playing with my little brother a lot, I just chase him around and around the house, and he, yeah, he enjoys that, and since he enjoys it, I enjoy it, and since I'm happy, Mom and Dad are happy. Yeah.

Bear likes to say when he has a great answer for something or something cool to tell you, "Put your hands on your head," which we do, and then he says, "'cause your mind's gonna be blown." And that's how I feel about Evrysdi in general. It really has blown my mind.

Bear was about four and a half when he started Evrysdi. We could start to see that having something that he could take at home, having something he could take orally, that as he became older and his own independent person, that was gonna be important, you know, play such a pivotal role in every part of his day.

We had heard about Evrysdi actually online from a friend who lives nearby. Has two children with SMA.

I did hesitate about switching to Evrysdi initially because we had finally gotten Bear to what felt like a stable and safe place, that the idea of changing anything felt really scary to me, and we did talk about it a lot, Kevin and I, and we brought it all up to the neurologist.

It was really important to have the at-home treatment option for Evrysdi, so something that we could kind of fold into the background of his life. So once we spoke to our neurologist about both options, she agreed and she had been very pivotal to our other treatment decisions.

Bear has his motor skills measured in standard testing. He does a whole slew of tests, and some of them you know, sitting up on his side. How far can he lift his arms? Can he get his hands to his mouth? Sit off the edge of something? Can he kick? Things like that. So a lot of PT testing, OT testing, being able to lift different weights until he gets to one that's too heavy. And I think that seeing those benchmarks kind of increase is really important to him. He loves being able to beat his old score and it's a lot of hard work, but you know, he gets through them each time and he's impressed by his own strength, which is important.

Yeah, so he has therapies all week, every week.

I've noticed that Bear has a lot of improved arm strength and hand strength. I've seen that in his ability to lift a cup to his mouth, his ability to write with different writing utensils, and his ability to grasp things tighter. And when people go to hand him something, you know, it doesn't just fall to his lap. He can actually take it, which has been a big change in his interaction, especially with other children. And that one's really important to him and me.

I can definitely hold myself up a lot easier. I feel like I've accomplished that.

He notices when he's able to do more things by himself, you know, thanks to treatments and getting a little bit stronger, he can get his own hand back on his joystick, which is a key piece because if he's driving around on his own, he wants to go around the other aisle of the grocery store, which is a big deal for him.

Like a seven-year-old, he loves video games, those little movements in his fingers to be able to play those games, like, it makes a huge difference. And also on his iPad, being able to reach things.

With Dad, I love playing video games with him. I love painting with Mommy.

When I think about giving Bear his treatment at home, I am always impressed. It feels kind of unbelievable that we went from really having no options to having a treatment that is in my refrigerator, that I can travel with, that he can take anywhere, that I can give it to him if he's taking a nap because it goes through his G-tube. And it's just amazing to me that something so small is having such an impact on his life.

For other families that are considering Evrysdi, of course, talk to your neurologist. I have to say, ask them anything and everything. They want to talk to you, they have the information, that is what they're there for, and don't be afraid of that. When I think about everything that Bear has been through, and how he was versus how he is today, I really am, really astounded.

So any parents that are concerned about or worried about side effects when it comes to Evrysdi, first I would say completely understandable. Whenever we've considered different treatment options for Bear, we've of course looked at all the side effects, so I think that looking at all of the options, doing as much research as possible, finding out what fits best in your lifestyle, is really important. And we've never noticed any side effects for him since the start of treatment really, so we've been looking at that.

For Bear's future, we're really just hoping that we can help kind of clear the way for him to be who he's already becoming, kind of, you know, just adventurous, and joyful and fun.

Bear has a great, strong personality and an amazing spirit, and I think we just hope that he takes that with him wherever he goes.

This is Evrysdi in action.

What is Evrysdi?

Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
- are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
- are a woman who can become pregnant:
  - Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
  - Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
  - Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting www.evrysdipregnancyregistry.com
- are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
- are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
Tell your healthcare provider about all the medicines you take
You should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
The most common side effects of Evrysdi include:
- For later-onset SMA:
  - fever
  - diarrhea
  - rash
- For infantile-onset SMA:
  - fever
  - diarrhea
  - rash
  - runny nose, sneezing, and sore throat (upper respiratory infection)
  - lung infection (lower respiratory infection)
  - constipation
  - vomiting
  - cough

These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

Please see full Prescribing Information for additional Important Safety Information.

- Información de prescripción de Evrysdi^® (risdiplam). Genentech, Inc.
  
  Información de prescripción de Evrysdi^® (risdiplam). Genentech, Inc.