Evrysdi, also known as risdiplam, is an oral medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to muscle weakness. Muscle weakness may affect normal everyday functions like head movement, sitting without help, walking, swallowing, or breathing. SMA is divided into types based on when symptoms appear. It may be called infantile if the symptoms first appear in infants, juvenile if symptoms first appear in children, or adult-onset, if symptoms first appear in adults.
Below you’ll find common questions and answers about Evrysdi.
How Is Evrysdi Taken?
When you get your Evrysdi from the specialty pharmacy, it should be in liquid form already. If your supply of Evrysdi is not mixed into a liquid, do not take your dose from the bottle and contact your pharmacist or health care provider.
Your health care provider will tell you what dose to use and show you how to measure and prepare it. Your bottle should come with a cap, a bottle adapter, and reusable syringes. If any of these parts are missing or broken, do NOT take your dose of Evrysdi until you contact your pharmacist or health care provider.
Your pharmacist or health care provider will show you how to draw up doses from the bottle and how to take them by mouth or by feeding tube. Keep all instructions for future reference.
You should use the reusable syringe that is given to you to measure your doses. Contact your health care provider if the original syringe provided is lost or damaged. You may wash and reuse the syringe for future doses.
Once you have drawn up your dose into the syringe, you should take it right away. If you do not take it within five minutes of drawing it up, throw it away and prepare a new dose.
You should take Evrysdi at the same time every day. Evrysdi should be taken after eating or after breastfeeding (for infants). Evrysdi should not be mixed with formula or milk. You should drink water after your dose to be sure it was completely swallowed.
Evrysdi can also be taken through a feeding tube. The tube should be flushed with water after the dose is pushed through.
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.
Do not mix liquids from different bottles to make a dose.
How Was Evrysdi Studied?
Evrysdi was studied in patients with infantile-onset (type 1 SMA), later-onset (type 2 or type 3 SMA), and in people with a diagnosis of SMA who did not show symptoms yet (pre-symptomatic SMA).
- Infantile-onset trial:
- Evrysdi was studied in a trial that included 58 infants who had type 1 SMA. Infants were about 6 months old when they started the trial.
- Later-onset trial:
- Evrysdi was studied in a trial of 180 children and adults with type 2 or type 3 SMA who were between 2 and 25 years of age.
- About two-thirds of the people in the trial received Evrysdi, and the other one-third received a placebo (containing no medicine).
- Presymptomatic trial:
- Evrysdi was studied in a trial that included 26 infants (up to 6 weeks of age) who were diagnosed with SMA based on their genes.
What Types of People Were in These Studies?
Here are some traits of people who took part in these studies.
- In the infantile-onset study, about 60% of infants in this trial were female. Also, 57% were Caucasian and 29% were Asian.
- About half of the people (51%) in the later-onset trial were female. Also, 67% of people were Caucasian and 19% were Asian. The average age at the start of the trial was 9 years, and 67% of people in the trial had scoliosis.
- Infants were about 25 days old when they received the first dose. A total of 62% of the infants in this trial were female and about 85% were Caucasian.
What Benefits Were Seen in These Studies?
- Infantile-onset trial. Of the infants who received Evrysdi for 12 months, about a third were able to sit without support for at least five seconds. After 24 months, about 60% were able to sit without support for at least five seconds.
- Later-onset trial. People who received Evrysdi for 12 months had improvements in their motor function score, and those who received a placebo had a decrease in their motor function score.
- Presymptomatic trial. At the end of the 12-month trial, 25 of the 26 infants (96%) treated with Evrysdi were able to sit without support for at least five seconds. Also, 21 of the 26 infants (about 81%) were able to sit without support for 30 seconds.
Your results may differ from what was seen in clinical studies.
What Are the Side Effects of Evrysdi, and How Can I Manage Them?
The most common side effects seen with Evrysdi are fever, diarrhea, and rash.
If you develop diarrhea, it’s important to make sure you are getting enough fluid. If you are feeding an infant, or breastfeeding, you may want to feed more regularly. Cereal is a good solid food option for infants on baby food. Consider oral rehydrating solutions, such as Pedialyte, and give 2 to 4 ounces after each watery stool.
These are not all of the possible side effects of Evrysdi. Talk with your health care provider if you are having symptoms that bother you. If you experience anything that you think may be caused by Evrysdi, you can also report side effects to the FDA at 800-FDA-1088 (800-332-1088).
Is It Safe to Take Evrysdi if I Am Pregnant?
Tell your health care provider if you are or plan to become pregnant. Your health care provider will advise you if you should take Evrysdi while you are pregnant or trying to get pregnant. Evrysdi may cause harm to an unborn baby.
A negative pregnancy test is needed before starting Evrysdi. If you can become pregnant, use effective birth control while taking Evrysdi and for at least one month after the last dose. If you become pregnant while taking Evrysdi, you can sign up for the Evrysdi registry by calling 833-760-1098 or by visiting www.evrysdipregnancyregistry.com. This registry collects safety information about the use of Evrysdi during pregnancy.
How Do I Get Evrysdi?
You will receive your Evrysdi from a specialty pharmacy. Your medication will come directly to your door each month from the specialty pharmacy. MySMA Support is a service offered through Genentech that can help coordinate the delivery of your medicine with the specialty pharmacy. You can contact the MySMA Support team at 833-387-9734.
Is There Any Cost Assistance Available?
There may be cost assistance to help you afford Evrysdi. You may have options even if you do not have health insurance. MySMA Support is a service that can help with insurance and coverage questions. Your health care provider can help you get started, or you can call 866-4ACCESS (866-422-2377) to discuss any of the options below.
- The Evrysdi Co-pay Program can help provide some cost assistance to certain patients with insurance.
- Information on foundations that can assist with copays are listed on the drugmaker’s website at https://www.evrysdi-hcp.com/financial-support/assistance-options.html#copaycard.
- The Genentech Patient Foundation can help with costs for certain patients who qualify.
