The most inclusive clinical trial program in SMA

The most inclusive clinical trial program in SMA

More than 490 people aged newborn to 60 years with presymptomatic, Type 1, 2, or 3 SMA*

Over 490 SMA patients ranging from newborns to 60 years old
  • Broad range of physical ability 
  • Able to walk† 
  • Unable to walk
  • Severe scoliosis
  • Joint contractures
  • Previous treatment with approved or investigational SMA medicines


EFFICACY is being studied in people aged 16 days to 25 years

SAFETY is being studied in people aged 16 days to 60 years

The first trial program in spinal muscular atrophy to include people older than 18 years, some of whom have severe scoliosis
The first trial program in spinal muscular atrophy to include people older than 18 years, some of whom have severe scoliosis


*The efficacy and safety of Evrysdi were established in 3 main studies. SUNFISH is a 2-part, placebo-controlled study in 231 adults and children aged 2 to 25 years with Type 2 or 3 SMA. FIREFISH is a 2-part, open-label study in 62 infants aged 2 to 7 months with Type 1 SMA. RAINBOWFISH is an ongoing, open-label study in 26 newborns younger than 6 weeks (at first dose). These newborns were genetically diagnosed with SMA and had not yet shown symptoms (presymptomatic SMA). A fourth study, JEWELFISH, is an ongoing, open-label safety study in 174 people aged 1 to 60 years with Type 1, 2, or 3 SMA that was previously treated with approved or investigational SMA medications.
In SUNFISH Part 1, 7 people were able to walk; in JEWELFISH, 16.

 A closer look at the trials

SUNFISH is a 2-part, placebo-controlled study

A study of Evrysdi® in adults and children with Type 2 or 3 SMA

adults and children with Type 2 or 3 SMA

Main measurement after 12 months:

  •  Change in motor function* with Evrysdi, compared with placebo

PART 1

51 adults and children
(2 to 24 years)

  • Explored the dose and safety of Evrysdi
  • Included 7 people who could walk

PART 2

180 adults and children
(2 to 25 years)

  • Measured the safety and effectiveness of the recommended dose of Evrysdi (in 120 people), compared with placebo (in 60 people)
  • Included 180 people who were not able to walk, 120 people with scoliosis (57 with severe scoliosis), and people with and without joint contractures
The majority of individuals in SUNFISH would not have qualified for prior SMA studies
The majority of individuals in SUNFISH would not have qualified for prior SMA studies

*Measured by the Motor Function Measure-32 Items (MFM-32) scale.
Adults and children not taking Evrysdi took a placebo, a substance that has no active medication and is often used in studies.

FIREFISH is a 2-part, open label study

A study of Evrysdi® in infants with Type 1 SMA

infants with Type 1 SMA

Main measurements after 12 months:

  • Sitting without support for at least 5 seconds*
  • Survival without permanent breathing support

 

FIREFISH is made up of 2 parts

PART 1

Explored the recommended dose of Evrysdi in 21 infants (aged 3 to 7 months)

PART 2

Measured the safety and effectiveness of Evrysdi in 41 infants (aged 2 to 7 months) at the recommended dose

58 infants (2 to 7 months)

who received the recommended dose of Evrysdi in Parts 1 and 2 were included in a pooled analysis that evaluated the effectiveness of Evrysdi

*Measured by Item 22 of the Bayley Scales of Infant and Toddler Development–Third Edition (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 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.

RAINBOWFISH is an ongoing, open-label study

A study of Evrysdi® in in newborns with presymptomatic SMA

newborns younger than 6 weeks (at first dose) with genetically diagnosed SMA who had not yet shown symptoms (presymptomatic)

Main measurement after 12 months:

  • Sitting without support for at least 5 seconds*

 

AT THE TIME INITIAL RESULTS WERE COLLECTED

18 infants
had enrolled in the study

  • These infants were younger than 6 weeks (between 16 and 40 days) at the time of first Evrysdi dose

6 infants
had received Evrysdi for at least 12 months and were included in the measurement of effectiveness

  • These infants had either 2 or 3 copies of the SMN2 gene
  • There were not enough study participants to evaluate the main effectiveness measurement

*Measured by Item 22 of the Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III) among at least 5 infants with 2 copies of the SMN2 gene and compound muscle action potential (CMAP) amplitude ≥1.5 mV at the start of the study. CMAP is a measure of muscle activity after the nerve that controls the muscle is stimulated.

JEWELFISH is an ongoing, open-label safety study

A study of Evrysdi® in adults, chidlren and infants
adults, children, and infants with Type 1, 2, or 3 SMA who have received previous treatment with approved or investigational SMA medications
  • An ongoing, open-label safety study in people who previously took a different SMA medication
  • Participants range from 1 to 60 years old
  • Includes people who are able to walk, and those who are not able to walk, as well as infants who can sit and those who can’t
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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.