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*
Participants enrolled across all studies included a range of attributes:
- 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 for an FDA approved treatment 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 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
Measurements after 12 months:
- Change in motor function* with Evrysdi, compared with placebo† (main measurement)
- Change in upper limb function‡ with Evrysdi compared with placebo† (additional measurement)
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.
‡Measured by the Revised Upper Limb Function scale.
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†
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
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 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 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
*All but 3 patients enrolled in JEWELFISH received previous treatment; these 3 patients were previously enrolled in a different trial but received placebo only.
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Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
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