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Results in infants as young as 2 months with Type 1 SMA

Results in infants as young as 2 months with Type 1 SMA

Infant patients

FIREFISH is a 2-part, open-label study of Evrysdi in 62 infants aged 2 to 7 months with Type 1 SMA. Part 1 explored the dose and safety of Evrysdi in 21 infants.  Part 2 measured the effectiveness and safety of Evrysdi in 41 infants. Fifty-eight infants aged 2 to 7 months who received the recommended dose of Evrysdi in Parts 1 and 2 were included in a pooled analysis.

Learn more about the trial

Changing the course of SMA by helping infants sit without support for at least 5 seconds

Of the infants taking the recommended dose of Evrysdi
Of the infants taking the recommended dose of Evrysdi
Infant sitting up
FIREFISH PART 2 (MAIN MEASUREMENT)
AFTER 1 YEAR
29% of infants

of infants (12/41) were able to sit without support for at least 5 seconds, as measured by BSID-III

FIREFISH PART 2 (MAIN MEASUREMENT)
AFTER 1 YEAR
29% of infants

of infants (12/41) were able to sit without support for at least 5 seconds, as measured by BSID-III

FIREFISH PARTS 1 AND 2
AFTER 1 YEAR
33% of infants

of infants (19/58) were able to sit without support for at least 5 seconds, as measured by BSID-III

 

FIREFISH PARTS 1 AND 2
AFTER 1 YEAR
33% of infants

of infants (19/58) were able to sit without support for at least 5 seconds, as measured by BSID-III

 

AFTER 2 YEARS
60% of infants

of infants (35/58) were able to sit without support for at least 5 seconds, as measured by BSID-III

AFTER 2 YEARS
60% of infants

of infants (35/58) were able to sit without support for at least 5 seconds, as measured by BSID-III

Infants with Type 1 SMA are typically not able to sit on their own without treatment
Infants with Type 1 SMA are typically not able to sit on their own without treatment


BSID-III stands for Bayley Scales of Infant and Toddler Develoment-Third Edition.

Remarkable achievement of key milestones not typically seen in infants without treatment with Type 1 SMA

Of the infants taking the recommended dose of Evrysdi
Infant sitting up
AFTER 2 YEARS
40% of infants

of infants (23/58) were able to sit without support for at least 30 seconds, as measured by BSID-III

 

AFTER 2 YEARS
40% of infants

of infants (23/58) were able to sit without support for at least 30 seconds, as measured by BSID-III

 

AFTER 2 YEARS
28% of infants

of infants (16/58) were able to stand, as measured by HINE-2

  • 9/58 could stand supporting weight
  • 7/58 could stand with support
AFTER 2 YEARS
28% of infants

of infants (16/58) were able to stand, as measured by HINE-2

  • 9/58 could stand supporting weight
  • 7/58 could stand with support

BSID-III stands for Bayley Scales of Infant and Toddler Development–Third Edition. HINE-2 stands for Hammersmith Infant Neurological Examination–Module 2.

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

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 has been the right decision for our family. Kayden has just had a wonderful response.”

Amanda, whose son Kayden is a little brother, greeting card lover, and master of silly noises, living with Type 1 SMA
Kayden, living with Type 1 SMA

Evrysdi slowed disease progression of SMA

Of the infants taking Evrysdi (all dose strengths)

 

Of the infants taking Evrysdi (all dose strengths)

 

lungs icon
AFTER 1 YEAR
87% of infants

of infants (54/62) were alive and able to breathe without permanent support*

AFTER 1 YEAR
87% of infants

of infants (54/62) were alive and able to breathe without permanent support*

AFTER 2 YEARS
84% of infants

of infants (52/62) were alive and able to breathe without permanent support*

AFTER 2 YEARS
84% of infants

of infants (52/62) were alive and able to breathe without permanent support*

Without treatment, infants with Type 1 SMA are not expected to survive beyond
14 months of age without permanent breathing support
Without treatment, infants with Type 1 SMA are not expected to survive beyond 14 months of age without permanent breathing 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 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.

Of the infants taking the recommended dose of Evrysdi
AFTER 1 YEAR

85% of infants (49/58) were able to eat by mouth*
86% of infants (50/58) were able to swallow†

AFTER 2 YEARS

83% of infants (48/58) were able to eat by mouth*
86% of infants (49/58) were able to swallow†

This information is considered exploratory. This means it was not designed to show a treatment effect so conclusions cannot be drawn.

*Includes infants who could eat by mouth or in combination with a feeding tube.
†One infant could not swallow at the start of the study.

Jewelfish trial icon
Learn more about JEWELFISH, an ongoing, 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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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. Talk to your healthcare provider right away if you become pregnant while taking Evrysdi. Ask about registering with the Evrysdi Pregnancy Registry, which was created to collect information about your health and your baby's health. Your healthcare provider can enroll you in this registry 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.