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Results in infants as young as 2 months

Results in infants as young as 2 months

Infant patients

FIREFISH is a 2-part, open-label clinical study in infants with Type 1 spinal muscular atrophy (SMA). It included 62 infants 2 to 7 months old.

Learn more about the trial

Evrysdi helped infants sit without support for at least 5 seconds

EVRYSDI IN INFANTS (FIREFISH PART 1)
Of the infants taking the recommended dose of Evrysdi
Of the infants taking the recommended dose of Evrysdi
Infant sitting up
AFTER 12 MONTHS
41% of infants sat without support

of infants (7/17) sat without support for at least 5 seconds, as measured by BSID-III

AFTER 12 MONTHS
41% of infants sat without support

of infants (7/17) sat 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.

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.

Exploratory observations: sitting without support for at least 5 seconds after 24 months
FIREFISH PART 1
AFTER 24 MONTHS

59% of infants (10/17) taking the recommended dose of Evrysdi sat without support for at least 5 seconds as measured by BSID-III

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

“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 helped infants survive and slowed disease progression

EVRYSDI IN INFANTS (FIREFISH PART 1)
Of the infants taking Evrysdi
Of the infants taking Evrysdi
lungs icon
AFTER 12 MONTHS
90% of infants were alive and able to breathe

of infants (19/21) were alive and able to breathe without permanent support*

 

AFTER 23 MONTHS

81% of infants (17/21) were alive and able to breathe without permanent support*

AFTER 12 MONTHS
90% of infants were alive and able to breathe

of infants (19/21) were alive and able to breathe without permanent support*

 

AFTER 23 MONTHS

81% of infants (17/21) 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 12 MONTHS

88% of infants (15/17) were able to eat by mouth*
88% of infants (15/17) were able to swallow†

AFTER 24 MONTHS

77% of infants (13/17) were able to eat by mouth*
82% of infants (14/17) 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.

*Two of these infants could eat some food by mouth, but also used 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 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, 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
    • 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, 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.

    • Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

      Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.