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.

Helping preserve daily function

EVRYSDI IN ADULTS AND CHILDREN (SUNFISH PART 2)

Change in motor function score over 1 year vs placebo
As measured by MFM-32

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 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.

CI stands for confidence interval. 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 enabled meaningful improvement of upper limb function

EVRYSDI IN ADULTS AND CHILDREN (SUNFISH PART 2)
Change in upper limb function score over 1 year vs placebo
As measured by RULM
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 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.

CI stands for confidence interval. RULM stands for Revised Upper Limb Module.

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

 

 

Hear from community members

questions icon

Questions?

We can help

hear from others icon

Explore community videos

Hear community perspectives

Video Player is loading.
Current Time 0:00
Duration 7:11
Loaded: 2.30%
Stream Type LIVE
Remaining Time 7:11
 
1x
    • Chapters
    • descriptions off, selected
    • captions off, selected
    • en (Main), selected
    1. Now Playing
      Up NextInspiring the next generation
    2. Now Playing
      Up NextTurning passion into purpose
    3. Now Playing
      Up NextPursuing justice for others
    4. Now Playing
      Up NextOwning the day
    5. Now Playing
      Up NextVoicing creativity

    Important Safety Information and Indication

    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
    • If you were prescribed Evrysdi for Oral Solution, 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.