Empowering understanding
Designed with your needs in mind, this video includes instructions on how to make Evrysdi part of your routine.
Get answers about Evrysdi and spinal muscular atrophy (SMA)
Evrysdi is the first and only oral, non-invasive, at-home treatment with results in adults, children, and infants with spinal muscular atrophy (SMA).
Spinal muscular atrophy (SMA) is caused by a shortage of the survival motor neuron (SMN) protein, which your muscles need to function. As a result, muscles throughout your body continuously weaken.
Evrysdi is a small molecule that is taken orally and designed to reach parts of the body that need SMN protein, such as the brain and spinal cord, muscles, larynx, GI system, and the heart.
*This was observed when Evrysdi was studied in animals.
Evrysdi was studied in the most inclusive clinical trial program for SMA. These studies included more than 490 people aged newborn to 60 years with Types 1, 2, 3, and presymptomatic SMA.
The most common side effects of Evrysdi in adults in children are fever, diarrhea, and rash. The most common side effects of Evrysdi in infants are fever, diarrhea, rash, runny nose, sneezing, sore throat, lung infection, constipation, vomiting, and coughing. These are not all the possible side effects of Evrysdi.
There are patient financial assistance resources for Evrysdi. MySMA Support™ is a support service from Genentech to help assist with insurance coverage and potential financial assistance options.
A PAL is a dedicated local Genentech representative who supports people living with SMA and their caregivers. A PAL can provide in-person or virtual support based on your preference and connect you to helpful resources. The MySMA SupportTM team, including the PAL, does not provide medical advice and is not a substitute for your medical team.
Your recommended dose of Evrysdi is based on age and weight, with a maximum recommended dose of 5 mg once daily. Your healthcare provider determines your dose. Always take your dose exactly as prescribed.
The missed dose should be taken as soon as possible, but no more than 6 hours after the usual dosing time. If more than 6 hours have passed, skip the missed dose altogether and take or give the next dose the following day at your regularly scheduled time.
Do not give any more medication that day. Wait until the next day to give the next dose at the usual dosing time. If your child continues to spit up or vomit while taking Evrysdi, talk to your healthcare provider.
Before starting treatment, tell your healthcare provider if you are taking any prescription medications (such as statins), over-the-counter medications (such as stool softeners), vitamins, dietary supplements, or additional fiber that may impact bowel movements, what foods you generally eat, as well as your typical meal times. Discuss if any part of your routine may need to be adjusted, including prescription medicines, dairy products, fatty foods, high-fiber foods, highly seasoned foods, or foods that have a laxative effect.
Questions about Evrysdi liquid
The medication lasts up to 64 days after being prepared by a pharmacist. An expiration date will be indicated on the bottle of Evrysdi. It may be helpful to mark that date on your calendar. If you receive a bottle without the Discard After date written on the bottle label, contact your specialty pharmacy.
Yes. Each monthly shipment of Evrysdi will include syringes that will work with a variety of feeding tubes, including ENFit®. If the specialty pharmacy does not provide the correct adapter to fit your feeding tube, they will work with you to get one that does.
If you or the person in your care will be away from home, store Evrysdi liquid in an insulated cooler (36°F to 46°F with ice packs), but do not freeze it. Store Evrysdi liquid upright in the original amber bottle to protect it from light. If traveling by plane, your healthcare provider may give you a letter authorizing you to fly with liquid medication. Talk to your healthcare provider about the best way to travel with Evrysdi liquid.
Evrysdi liquid can be kept at room temperature up to 104°F (40°C) for a combined total of 5 days. It can be removed from, and returned to, a refrigerator. The total combined time out of refrigeration should not exceed 5 days. Any portion of Evrysdi liquid that has been kept above 104°F (40°C) should be discarded. If you have any questions regarding storing Evrysdi liquid at room temperature, contact your specialty pharmacy.
Evrysdi should not be mixed directly with any food or liquid (including formula or breast milk), and should only be taken with the approved oral syringe, which has been designed to protect the solution from light. You or the person in your care should drink some water (about 1 tablespoon) after swallowing the medication to ensure all the medication is swallowed. If you have questions, speak to your healthcare provider.
Questions about Evrysdi tablets
Evrysdi tablets are approved for people aged 2 years and older who weigh at least 44 lb (20 kg). The tablets cannot be taken with feeding tubes.
Evrysdi tablets can either be swallowed whole with water or as a mixture with at least 1 teaspoon (5 mL) of room temperature non-chlorinated drinking water (eg, filtered water, bottled water). They cannot be chewed, crushed, cut, or dissolved on the tongue.
To help make sure every dose is consistent, always mix it with bottled water. Chlorine in tap water can interact with the medicine in Evrysdi.
Your ability to switch between formulations will depend on discussions with both your healthcare and insurance providers. Be sure to ask your healthcare provider which option is best for you.
Evrysdi tablets are 6.6 mm (0.25 in) in diameter and 4.1 mm (0.15 in) in height—about the size of a kernel of corn.
Your insurance company may require you to use all of your current medicine before you begin taking Evrysdi tablets. Be sure to check with your insurance company to be certain.
Evrysdi tablets can be taken with or without food. It will not affect how the tablet works either way. Just remember to take it at approximately the same time each day.
No, unfortunately it cannot be administered via a gastrostomy tube (G-tube) or a nasogastric tube (NG-tube). If you or the person you are caring for has an G-tube or an NG-tube, you can ask your healthcare provider about taking the Evrysdi liquid.
If you have concerns about costs or insurance coverage, our PALs (Partnership and Access Liaisons) are here to help. You can visit Evrysdi.com/PAL to learn more and connect with a PAL. Reach out anytime.
Evrysdi tablets are only available through a specialty pharmacy. Once prescribed, they will be shipped directly to your door.
The film coating on the tablet protects patients from the active ingredients until they are ingested into the stomach by eating or drinking, and processed. Chewing, crushing, or otherwise breaking the film coating may result in extended exposure to the active ingredients before ingestion. The safety of Evrysdi tablets was studied when swallowed whole with water and when dispersed with bottled water. The safety of taking Evrysdi tablets any other way is unknown and therefore is not recommended.
No. To avoid exposing the tablets to moisture, keep the tablets in their original container with the desiccant (non-edible substance) in the cap.
Request helpful tools and resources
Financial assistance is available
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Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
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Atrophy
The medical term for getting smaller, which is generally what happens to muscles when they are not stimulated by nerve cells.
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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.
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CI
Stands for confidence interval, which shows how much a clinical result is likely to vary.
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DMT
Stands for disease-modifying treatment, which in the case of SMA, stimulates production of SMN2 protein.
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FDA
Stands for Food and Drug Administration.
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G-tube
Stands for gastrostomy tube, which is a tube inserted through the belly that brings nutrition directly to the stomach.
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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.
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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.
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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.
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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.
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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.
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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.
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Molecule
The smallest particle of a substance, made up of one or more atoms.
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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.
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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.
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Oral
Refers to anything having to do with the mouth.
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PAL
Stands for Partnership and Access Liaison.
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Presymptomatic
When someone has not yet displayed any symptoms but may have underlying presence of the disease, as shown through testing or other means.
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Previously treated
When someone has taken either an approved or investigational medication specifically for SMA in the past.
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Progression
When the symptoms of SMA get worse over time.
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Progressive disease
Any condition that gets worse over time instead of improving.
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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.
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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.
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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.
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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.
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SMA
Stands for spinal muscular atrophy, which is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement.
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SMN
Stands for survival motor neuron, which is a type of protein that your muscles need to function.
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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.
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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.
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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.
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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.