Evrysdi is being studied in a broad range of infants, children, and adults with SMA

Including more than 450 people with Type 1, 2, or 3 spinal muscular atrophy (SMA)

2 months to 60 years. Efficacy is only being studied in people from 2 months to 25 years old with the following characteristics. Those with varying symptoms and motor functions. Those who could or could not walk. Those without scoliosis to those with severe scoliosis or joint contractures. Those who have or have not taken medication for SMA before. This is the first time a medication for SMA is being studied in people over 18 years of age, some of whom have severe scoliosis

Evrysdi received FDA approval based on results from the first 12 months of 2 main ongoing studies

FIREFISH An open-label, 2-part study of infants with Type 1 SMA

FIREFISH is an open-label study of 62 infants made up of 2 parts

2 months of age to 7 months of age. Measured the ability of infants to sit without support for at least 5 seconds and survival without permanent breathing support after 12 months of taking Evrysdi®️ (risdiplam)

PART 1

  • Includes 21 infants
  • Explored the recommended dose of medication for people in this age group
  • Measured the safety and effectiveness of Evrysdi after 12 months

PART 2

  • Includes 41 infants
  • Is measuring the safety and effectiveness of Evrysdi at the recommended dose
  • Part 2 is ongoing

See results in infants with Type 1 SMA

SUNFISH A randomized, placebo-controlled, 2-part study of children and adults with Type 2 or 3 SMA

SUNFISH is a randomized, placebo-controlled study of 231 children and adults made up of 2 parts

2 years of age to 25 years of age. Measured the change in motor function after 12 months of treatment, compared with those not taking Evrysdi®️ (risdiplam)
PART 1
  • Includes 51 children and adults
  • Explored the recommended dose of medication for people in this age group
  • Is exploring the safety of Evrysdi for people in this age group
  • Includes 7 people who could walk

PART 2

  • Includes 180 people
  • Is measuring the safety and effectiveness of Evrysdi at the recommended dose (n=120), compared with those not taking Evrysdi (n=60)
  • Includes:
    • Those with scoliosis (n=120), sometimes severe (n=57)
    • Those with or without contractures
    • Those who cannot walk (n=180)

Measured by Item 22 of the BSID-III Gross Motor Scale.
Permanent support was defined as having a tracheostomy (a surgery where a tube is inserted in the front of the throat into the windpipe) or more than 21 days of noninvasive ventilation support (16 or more hours a day) or being intubated (a procedure where a breathing tube is inserted down the throat and into the windpipe) to help with breathing in the absence of an acute reversible event.
§Measured by the MFM-32 scale.
||Those not taking Evrysdi were on placebo, a substance that has no active medication and is often used in studies.

FDA stands for the US Food and Drug Administration

See results in children and adults with Type 2 or 3 SMA

A third, supportive study is currently looking at the safety of Evrysdi in people previously treated for SMA

JEWELFISH An ongoing study to monitor safety for people with Type 1, 2, or 3 SMA who were previously treated with other SMA medications
  • Includes 174 people who had taken other SMA medications before 
  • From 1 to 60 years old
  • This study is ongoing

*Motor function was measured using several tools including the Bayley Scales of Infant and Toddler Development Gross Motor Scale-Third Edition (BSID-III), the Motor Function Measure-32 Items (MFM-32), and the Revised Upper Limb Module test (RULM).

MySMA Support


Learn more about Evrysdi, financial support options, prescription shipment, and more

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.