Results in adults and children

Results in adults and children

Jim, living with Type 3 SMA

Adults and children significantly 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.

Evrysdi significantly improved or maintained 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 image

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.

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

My improved motor function with Evrysdi has made a real difference. I have noticed changes in arm strength, I can pick up small objects and open containers with greater ease, and I’ve been working with my physical therapist to do more movements with my legs.”

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

Evrysdi significantly improved strength in arm and hand movements vs placebo

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.

“With Evrysdi, I’ve noticed real improvements – plus, it’s an oral treatment I can take at home!

Christa, artist, therapy-dog trainer, and social butterfly living with Type 2 SMA
Christa, artist, therapy dog trainer, living with Type 2 SMA

 

 

Hear from community members

Jim in action

Hear from Jim about his experience on Evrysdi

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 voiceover 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 some 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.

Dan in action

Hear from Dan about his experience on Evrysdi

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.

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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
    • 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.