Evrysdi is a disease-modifying therapy approved to treat SMA

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.

Evrysdi®️ (risdiplam) is designed to work by helping to make and maintain more SMN protein

In animal studies, Evrysdi was distributed throughout the body

Evrysdi increased and maintained levels of SMN protein

Within 4 weeks of starting Evrysdi®️ (risdiplam), median SMN protein levels more than doubled and were maintained throughout 12 months of studies across all SMA types

Evrysdi received FDA approval based on results from the first 12 months of 2 main ongoing studies

FIREFISH Infants with Type 1 SMA SUNFISH Children and adults with Type 2 or 3 SMA

A third, supportive safety study, JEWELFISH, is currently looking at people previously treated for SMA.

The studies of Evrysdi include people with Type 1, 2, or 3 SMA from 2 months to 60 years old

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

41% of infants sat without support for at least 5 seconds. 7 out of 17 infants.

Untreated infants with Type 1 SMA are not expected to sit without support

90% of infants (19 out of 21) at 12 months and 81% of infants (17 out of 21) at 23 months were alive and could breathe without permanent support

See results in infants with Type 1 SMA

In SUNFISH Part 2 (Types 2 & 3 SMA) 

Motor function improved in children and adults taking Evrysdi®️ (risdiplam) after 12 months (average 1.36 point increase on the MFM-32 scale compared with an average 0.19 point decrease for those not taking Evrysdi)

See results in children and adults with Type 2 or 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 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 

A liquid taken once a day by mouth or feeding tube. Delivered directly to you.

The MySMA Support team can answer additional questions about Evrysdi and prescription delivery

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.

Evrysdi-At-A-Glance

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

What is Evrysdi?

Evrysdi® (risdiplam) is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.

 

It is not known if Evrysdi is safe and effective in children under 2 months of age.

  • Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
    • are pregnant or plan to become pregnant. If you are pregnant, or are planning to become pregnant, ask your healthcare provider for advice before taking this medicine. Evrysdi may harm your unborn baby.
    • are a woman who can become pregnant:
      • Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy. Because Evrysdi may harm your unborn baby, your healthcare provider will decide if taking Evrysdi is right for you during this time
      • Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
    • are an adult male planning to have children: Evrysdi may affect a man’s ability to have children (fertility). If this is of concern to you, make sure to ask a healthcare provider for advice
    • are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby. If you plan to breastfeed, discuss with your healthcare provider about the best way to feed your baby while on treatment with Evrysdi
  • Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine
  • You should receive Evrysdi from the pharmacy as a liquid that can be given by mouth or through a feeding tube. The liquid solution is prepared by your pharmacist. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
  • Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
  • The most common side effects of Evrysdi include:
    • For later-onset SMA:
      • fever
      • diarrhea
      • rash
    • For infantile-onset SMA:
      • fever
      • diarrhea
      • rash
      • runny nose, sneezing, sore throat, and cough (upper respiratory infection)
      • lung infection
      • constipation
      • vomiting

These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.


You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.


Please see full Prescribing Information for additional Important Safety Information.