After taking Evrysdi, motor function improved in children and adults

Key measurements for children and adults with Type 2 or 3 spinal muscular atrophy (SMA) in SUNFISH Part 2

Motor function

As measured by MFM-32

Children and adults treated with Evrysdi showed improved motor function compared with those not taking Evrysdi*

Average score increased 1.36 points for those treated with Evrysdi for 1 year (n=115). Average score decreased 0.19 points for those not taking Evrysdi®️ (risdiplam)  for 1 year (n=59)

Children and adults treated with Evrysdi showed improved motor function at 12 months

Change in motor function measured over 12 months. Average change in motor function from start of study on MFM-32 scale over 12 months. 1.36-point increase with Evrysdi (n=115). 0.19-point decrease without Evrysdi®️ (risdiplam) (n=59). Talk to your healthcare provider about setting treatment goals specific to you. Goals may vary based on factors like your age and current motor function abilities.

Exploratory observations: results after 24 months

Change in motor function score over 24 months

*Those not taking Evrysdi were on 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.
In some studies, including this one, if someone’s data cannot be collected on time for any reason, their progress cannot be counted in that part of the study. This chart only includes the information that was collected on time.

Upper limb function

As measured by the Revised Upper Limb Module (RULM) assessment

Children and adults treated with Evrysdi showed greater improvements in upper limb function compared with those who were not taking Evrysdi*

Change in upper limb score over 12 months. Average change from start of the study in RULM total score over 12 months. 1.61-point increase with Evrysdi®️ (risdiplam) (n=119). 0.02-point increase without Evrysdi (n=58).

The RULM scale measures upper limb strength by evaluating the performance of daily tasks, like:

Picking up coins/small objects. Using a pencil. Raising a cup to mouth. Opening a plastic container.

Exploratory observations: results after 24 months

Change in upper limb score over 24 months

*Those not taking Evrysdi were on 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.

In some studies, including this one, if someone’s data can’t be collected on time for any reason, their progress cannot be counted in that part of the study. This chart only includes the information that was collected on time.

Measurement tools

More about the MFM-32 scale

The Motor Function Measure-32 Items (MFM-32) scale is designed to capture change for a broad range of people, including those who can and cannot walk. It evaluates 32 different elements to assess head and trunk motor movement. The elements are grouped into 3 categories of movements, including standing/transfers, upper/lower body, and hand/foot.

MFM-32 measures motor function abilities that relate to important daily functions. The scale includes motor movement across 3 categories:
Standing/transfer movements. Upper/lower body movements. Hand/foot movements
Talking with your healthcare provider

Use these questions to guide your discussion about Evrysdi with your healthcare provider

What is Evrysdi?

Evrysdi® (risdiplam) is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.

 

It is not known if Evrysdi is safe and effective in children under 2 months of age.

  • Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
    • are pregnant or plan to become pregnant. If you are pregnant, or are planning to become pregnant, ask your healthcare provider for advice before taking this medicine. Evrysdi may harm your unborn baby.
    • are a woman who can become pregnant:
      • Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy. Because Evrysdi may harm your unborn baby, your healthcare provider will decide if taking Evrysdi is right for you during this time
      • 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 planning to have children: Evrysdi may affect a man’s ability to have children (fertility). If this is of concern to you, make sure to ask a healthcare provider for advice
    • are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby. If you plan to breastfeed, discuss with your healthcare provider about the best way to feed your baby while on treatment with Evrysdi
  • Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine
  • You should receive Evrysdi from the pharmacy as a liquid that can be given by mouth or through a feeding tube. The liquid solution is prepared by your pharmacist. 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, sore throat, and cough (upper respiratory infection)
      • lung infection
      • constipation
      • vomiting

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.