Evrysdi is being studied in a broad range of infants, children, and adults with SMA

Including more than 450 people with Type 1, 2, or 3 spinal muscular atrophy (SMA)

2 months to 60 years. Efficacy is only being studied in people from 2 months to 25 years old with the following characteristics. Those with varying symptoms and motor functions. Those who could or could not walk. Those without scoliosis to those with severe scoliosis or joint contractures. Those who have or have not taken medication for SMA before. This is the first time a medication for SMA is being studied in people over 18 years of age, some of whom have severe scoliosis

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

FIREFISH An open-label, 2-part study of infants with Type 1 SMA

See the difference Evrysdi made for infants with SMA. Type 1 SMA Results button

SUNFISH A randomized, placebo-controlled, 2-part study of children and adults with Type 2 or 3 SMA

See the difference Evrysdi made for children and adults with SMA. Type 2 and 3 SMA Results button

A third, supportive study is currently looking at the safety of Evrysdi in people previously treated for SMA

JEWELFISH An ongoing study to monitor safety for people with Type 1, 2, or 3 SMA who were previously treated with other SMA medications

*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).

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What is Evrysdi?

Evrysdi^TM (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:
- have liver problems
- 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.

Evrysdi is being studied in a broad range of infants, children, and adults with SMA

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

FIREFISH is an open-label study of 62 infants made up of 2 parts

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

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

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Important Safety Information & Indication

What is Evrysdi?