US Healthcare Professionals

How Evrysdi was studied

Evrysdi was proven safe and effective across SMA types*

Studied in the most inclusive clinical trial program in spinal muscular atrophy (SMA)

FDA APPROVED SINCE 2020

Studied in
4
Clinical trials

including
490+
people

people aged

16

days

–

60

years

with presymptomatic and Types 1, 2, and 3 SMA†

Participants enrolled across all studies included a range of attributes:

broad range of physical ability

able to walk‡

unable to walk

severe scoliosis

joint contractures

previous treatment with approved or investigational SMA medications

*Types 1, 2, 3, and presymptomatic SMA. 
†RAINBOWFISH is an open-label study in 26 newborns younger than 6 weeks (at first dose). These newborns were genetically diagnosed with SMA and had not yet shown symptoms (presymptomatic SMA). FIREFISH is a 2-part, open-label study in 62 infants aged 2 to 7 months with Type 1 SMA. SUNFISH is a 2-part, placebo-controlled study in 231 adults and children aged 2 to 25 years with Type 2 or 3 SMA. JEWELFISH is an open-label safety study in 174 people aged 1 to 60 years with Type 1, 2, or 3 SMA that were previously treated with approved or investigational SMA medications.
‡In SUNFISH Part 1, 9 people were able to walk; in JEWELFISH, 15.

See additional details and results from each study:

Adults and children with Types 2 and 3 SMA
Infants with Type 1 SMA
Newborns with presymptomatic SMA
Adults and children with previously treated Types 1, 2, and 3 SMA
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A quick summary of Evrysdi

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Important Safety Information and Indication

What is Evrysdi?

Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

  • Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
    • are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
    • are a woman who can become pregnant:
      • Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
      • 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
      • Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com.
    • are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
    • are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
  • Tell your healthcare provider about all the medicines you take
  • If you were prescribed Evrysdi for Oral Solution, you should receive Evrysdi from the pharmacy as a liquid. 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, and sore throat (upper respiratory infection)
      • lung infection (lower respiratory infection)
      • constipation
      • vomiting
      • cough

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.

    • Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

      Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

    • Atrophy

      The medical term for getting smaller, which is generally what happens to muscles when they are not stimulated by nerve cells.

    • BSID-III

      Stands for the Bayley Scales of Infant and Toddler Development–Third Edition, which is a tool used to assess a range of physical abilities, such as sitting, rolling, and crawling.

    • CI

      Stands for confidence interval, which shows how much a clinical result is likely to vary.

    • DMT

      Stands for disease-modifying treatment, which in the case of SMA, stimulates production of SMN2 protein.

    • FDA

      Stands for Food and Drug Administration.

    • G-tube

      Stands for gastrostomy tube, which is a tube inserted through the belly that brings nutrition directly to the stomach.

    • Gene

      The basic unit of heredity passed from parent to child, made up of sequences of DNA (deoxyribonucleic acid). They contain information that leads to the development of physical traits such as hair color.

    • GI system

      Stands for gastrointestinal system, also called the digestive system, which refers to the group of organs that take in food and liquids and break them down into energy the body can use.

    • HINE-2

      Stands for Hammersmith Infant Neurological Examination–Module 2, which is a tool used to assess 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

    • Investigational

      Term used to describe medical treatments that are currently being studied in clinical trials to determine how well they work and how safe they are to take.

    • Larynx

      Also known as the voice box, is a hollow tube in the middle of the neck, above the windpipe and esophagus. The larynx allows you to breathe and make sounds.

    • MFM-32

      Stands for Motor Function Measure-32 Items, which is a tool designed to capture changes in head, trunk, and limb motor movements using 32 elements in a broad range of people, including those who cannot walk. It uses 3 main categories: standing/transfer movements, upper/lower body movements, and hand/foot movements.

    • Molecule

      The smallest particle of a substance, made up of one or more atoms.

    • NG-tube

      Stands for nasogastric tube, which is a thin, soft tube that goes in through the nose, down the throat, and into the stomach to provide nutrition or medicine to those who cannot consume by mouth.

    • Non-invasive

      Term used to describe medicines or medical procedures that do not require inserting anything (such as a needle) through the skin or into a body opening.

    • Oral

      Refers to anything having to do with the mouth.

    • PAL

      Stands for Partnership and Access Liaison.

    • Presymptomatic

      When someone has not yet displayed any symptoms but may have underlying presence of the disease, as shown through testing or other means.

    • Previously treated

      When someone has taken either an approved or investigational medication specifically for SMA in the past.

    • Progression

      When the symptoms of SMA get worse over time.

    • Progressive disease

      Any condition that gets worse over time instead of improving.

    • Protein

      Large, complex molecules that play many important roles in the body, including making up the structure of cells and ensuring proper function of tissues and organs.

    • RULM

      Stands for Revised Upper Limb Module, which is a tool specifically designed for people 2.5 years and older living with SMA to evaluate strength in arm movements and the ability to perform certain tasks. It includes tests, such as picking up objects, like coins/tokens, tearing paper, raising a cup to the mouth, and opening a plastic container.

    • Scoliosis

      A sideways curve of the spine. Instead of appearing straight, the spine of a person with scoliosis will have an abnormal S or C shape that leads to their hips or shoulders appearing uneven.

    • Sedation

      A state of calmness, relaxation, or sleepiness as a result of medications called sedatives. Sedation can be used to help you relax or fall asleep for a medical procedure.

    • SMA

      Stands for spinal muscular atrophy, which is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement.

    • SMN

      Stands for survival motor neuron, which is a type of protein that your muscles need to function.

    • Spinal cord

      A tube of tissue that runs from your brain down to your lower back and acts as an important part of your nervous system. The spinal cord carries nerve signals from your brain to the rest of your body, which allow you to feel sensations such as pressure or pain.

    • Type 1 SMA

      SMA symptoms that are present at 6 months of age or earlier. This is sometimes called “infantile onset.” Type 1 SMA is characterized by generalized muscle weakness, a weak cry, and muscle distress, often accompanied by failure to meet developmental milestones such as sitting up unassisted.

    • Type 2 SMA

      SMA symptoms that appear between the ages of 6 to 18 months. People with Type 2 SMA cannot stand or walk without help, but they can sit without support.

    • Type 3 SMA

      SMA symptoms that appear after 18 months of age. People with Type 3 SMA can walk independently but may have difficulty doing so or performing other movements such as running, rising from a chair, or climbing stairs.