How Evrysdi is designed to work in spinal muscular atrophy
In animal studies, Evrysdi was distributed throughout the body
Evrysdi increased and maintained levels of SMN protein
Evrysdi received FDA approval based on results from the first 12 months of 2 main ongoing studies
A third, supportive safety study, JEWELFISH, is currently looking at people previously treated for SMA.
Evrysdi is proven to make a difference in infants, children, and adults with SMA
- In people 2 months and older who have a broad range of symptoms and motor function
In FIREFISH Part 1 (Type 1 SMA), out of the infants taking the recommended dose of Evrysdi for 12 months
Untreated infants with Type 1 SMA are not expected to sit without support
In SUNFISH Part 2 (Types 2 & 3 SMA)
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.
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 the first and only medication to treat SMA with at-home dosing
Talk to your healthcare provider to learn more about Evrysdi.
*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 two halves.
†Measured by Item 22 of the BSID-III Gross Motor Scale.
‡Those not taking Evrysdi were on placebo, a substance that has no active medication and is often used in studies.
§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.