Evrysdi improved motor function scores in 2 main studies
Important Safety Information
Before taking Evrysdi, tell your doctor if you have liver problems, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. Evrysdi may harm an unborn or breastfed baby. Evrysdi may affect a man’s ability to have children (fertility). Tell your doctor about all the medicines you take.
These are not all the possible side effects of Evrysdi. Please refer to additional Important Safety Information below.
Evrysdi is being studied in more than 450 people with Type 1, 2, or 3 SMA, ages 2 months to 60 years
Efficacy was only studied in people from 2 months to 25 years old
FIREFISH is a 2-part study of infants with Type 1 SMA. Part 1
includes 21 infants (3-7 months). It explored the recommended dose of
Evrysdi for this age group. The study also measured safety and
effectiveness of Evrysdi. The main measurement was the ability of
infants to sit without support for at least 5 seconds and survival
without permanent breathing support after 12 months of taking Evrysdi.
SUNFISH is a 2-part study of children and adults with SMA. Part 2
includes 180 people and is measuring the safety and effectiveness of
Evrysdi at the recommended dose (n=120), compared with those not
taking Evrysdi (n=60). The main measurement was the change in motor
function after 12 months of treatment, compared with those not taking
Evrysdi. This includes 128 people with Type 2 SMA and 52 with Type 3
SMA. A third, supportive study, JEWELFISH, is looking at the safety of
Evrysdi in 174 people with Type 1, 2, or 3 SMA from 1 to 60 years old
who were previously treated with other SMA medications.
Overview of Evrysdi
In animal studies, Evrysdi was distributed throughout the body.
Within 4 weeks of starting Evrysdi, median|| SMN protein
levels more than doubled and were maintained throughout 12 months of
The first and only medication to treat SMA with at-home dosing
Evrysdi is a liquid treatment, taken by mouth or feeding tube, that
Talk to your healthcare provider about whether treatment with
Evrysdi is right for you
*The infants in the study ranged in age from 3 to
7 months at the start of treatment, but many were closer to 7
†Measured by Item 22 of the Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III).
‡Permanent support was defined as having a tracheostomy or more than 21 days of noninvasive ventilation support (16 or more hours a day) or being intubated to help with breathing in the absence of an acute reversible event.
§Those not taking Evrysdi were on placebo, a substance that has no active medication and is often used in studies.
||A median is the middle value of a set of data that have been put into numerical order. The median is the value that divides the data into 2 halves.
MFM-32 stands for Motor Function Measure-32 Items.