The most inclusive clinical trial program in SMA*
The most inclusive clinical trial program in SMA*
More than 450 people aged 2 months to 60 years with Types 1, 2, and 3 SMA
- Broad range of physical ability
- Able to walk†
- Unable to walk
- Severe scoliosis
- Joint contractures
- Previous treatment with approved or investigational SMA medicines
EFFICACY was studied in people aged 2 months to 25 years
SAFETY is being studied in people aged 2 months 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
*FIREFISH is a 2-part, open-label study in 62 infants aged 2 to 7 months with Type 1 SMA. SUNFISH is a 2-part, placebo-controlled study in 231 adults and children aged 2 to 25 years with Type 2 or 3 SMA. 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†
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 contracture
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
infants with Type 1 SMA
Main measurements after 12 months:
- sitting without support for at least 5 seconds*
- survival without permanent breathing support†
PART 1
21 infants
(3 to 7 months)
- Explored the recommended dose of Evrysdi for people in this age group
- Also measured the safety and effectiveness of Evrysdi after 12 months
PART 2
41 infants
(2 to 7 months)
- Measured the safety and effectiveness of Evrysdi at the recommended dose
*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.
JEWELFISH is an ongoing, open-label safety study
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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Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
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