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Results in newborns with presymptomatic SMA

Results in newborns with presymptomatic SMA

Evrysdi®️ (risdiplam) newborns living with SMA

A closer look at the RAINBOWFISH study

RAINBOWFISH is an open-label study in newborns younger than 6 weeks (at first dose) with genetically diagnosed, presymptomatic SMA or infants who have been diagnosed but had not yet shown symptoms.

Learn more about the trial

 

Evrysdi helped infants with a wide range of SMA severity sit without support

This was measured by a scale called BSID-III

AFTER 1 YEAR OF TREATMENT
INFANTS WITH 2 SMN2 COPIES (n=8)

of infants (7/8) were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

AFTER 1 YEAR OF TREATMENT
INFANTS WITH 2 SMN2 COPIES (n=8)

of infants (7/8) were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22
 

AFTER 1 YEAR OF TREATMENT
TOTAL INFANTS IN STUDY (N=26)

of infants (25/26) were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

  • 81% of infants (21/26) were able to sit without support for at least 30 seconds, as measured by BSID-III, Item 26

 

AFTER 1 YEAR OF TREATMENT
TOTAL INFANTS IN STUDY (N=26)

of infants (25/26) were able to sit without support for at least 5 seconds
As measured by BSID-III, Item 22

  • 81% of infants (21/26) were able to sit without support for at least 30 seconds, as measured by BSID-III, Item 26

 

The Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III) gross motor scale assesses a range of physical abilities such as sitting, rolling, and crawling.

Evrysdi helped infants sit, stand, or walk independently

This was measured by another scale called HINE-2

AFTER 1 YEAR OF TREATMENT
TOTAL INFANTS IN STUDY (N=26)*

of infants (24/25) were able to sit

AFTER 1 YEAR OF TREATMENT
TOTAL INFANTS IN STUDY (N=26)*

of infants (24/25) were able to sit

 

of infants (21/25) were able to stand

  • 13/25 could stand without support
  • 8/25 could stand with support

of infants (21/25) were able to stand

  • 13/25 could stand without support
  • 8/25 could stand with support

 

of infants (12/25) were able to walk independently

of infants (12/25) were able to walk independently

*1 infant could not be assessed for HINE-2 at the 1-year visit.

The Hammersmith Infant Neurological Examination– Module 2 (HINE-2) assesses 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

Evrysdi helped infants survive

AFTER 1 YEAR OF TREATMENT
100% of infants

of infants (26/26) were alive and able to breathe without permanent support*

AFTER 1 YEAR OF TREATMENT
100% of infants

of infants (26/26) were alive and able to breathe without permanent support*

*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 either non-invasive 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.

EXPLORATORY OBSERVATIONS SUGGEST

Presymptomatic infants taking Evrysdi were able to feed orally and swallow

AFTER 1 YEAR OF TREATMENT
TOTAL INFANTS IN STUDY (N=26)

of infants (25/26) were able to exclusively feed orally*

AFTER 1 YEAR OF TREATMENT
TOTAL INFANTS IN STUDY (N=26)

of infants (25/26) were able to exclusively feed orally*

 

AFTER 1 YEAR OF TREATMENT

of infants (26/26) were able to swallow

AFTER 1 YEAR OF TREATMENT

of infants (26/26) were able to swallow

This information is considered exploratory, which means the clinical study was not specifically designed to show a treatment effect on feeding and swallowing. Data should be interpreted with caution.

*1 infant was not assessed.

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
Safety
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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
  • 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.