Including more than 450 people with Type 1, 2, or 3 spinal muscular atrophy (SMA)
Evrysdi received FDA approval based on results from the first 12 months of 2 main ongoing studies
FIREFISH: infants with Type 1 SMA
PART 1
- Includes 21 infants
- Explored the recommended dose of medication for people in this age group
- Measured the safety and effectiveness of Evrysdi after 12 months
PART 2
- Includes 41 infants
- Is measuring the safety and effectiveness of Evrysdi at the recommended dose
- Part 2 is ongoing
SUNFISH: Children and adults with Type 2 or 3 SMA
SUNFISH is a randomized, placebo-controlled study of 231 children and adults made up of 2 parts
- Includes 51 children and
adults
- Explored the recommended dose of medication for people in this age group
- Is exploring the safety of Evrysdi for people in this age group
- Includes 7 people who could walk
PART 2
- Includes 180 people
- Is measuring the safety and effectiveness of Evrysdi† at the recommended dose (n=120), compared with those not taking Evrysdi (n=60)
- Includes:
- Those with scoliosis (n=120), sometimes severe (n=57)
- Those with or without
contractures
- Those who cannot walk (n=180)
†Measured by Item 22 of the 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 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.
§Measured by the MFM-32
scale.
||Those not taking Evrysdi were on placebo,
a substance that has no active medication and is often used in studies.
FDA stands for the US Food and Drug Administration
A third, supportive study is currently looking at the safety of Evrysdi in people previously treated for SMA
JEWELFISH: Safety in people previously treated for SMA
- Includes 174 people who had taken other SMA medications before
- From 1 to 60 years old
- This study is ongoing
*Motor function was measured using several tools including
the Bayley Scales of Infant and Toddler Development Gross Motor
Scale-Third Edition (BSID-III), the Motor Function Measure-32 Items
(MFM-32), and the Revised Upper Limb Module test (RULM).
MySMA Support
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