How Evrysdi is designed to work in spinal muscular atrophy (SMA)

SMA is a genetic condition caused by the shortage of a key protein called SMN, or survival motor neuron, which is needed for nerves and muscles to function properly.

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.


Get to know Evrysdi with an overview of how it works and how it could help