Evrysdi (Risdiplam) - Oral

What Is Evrysdi?

Evrysdi (risdiplam) is a prescription medication used to treat spinal muscular atrophy (SMA) in adults and children. Evrysdi belongs to a group of drugs called survival motor neuron 2 (SMN2) splicing modifiers. Evrysdi works by increasing the production of an essential protein called survival motor neuron protein. People with SMA have trouble making this protein; low levels can cause muscle weakness and breakdown.

Evrysdi is available as an oral solution. 

Drug Facts

Generic Name: Risdiplam

Brand Name(s): Evrysdi

Drug Availability: Prescription

Therapeutic Classification: Survival motor neuron 2 (SMN2) spicing modifier

Available Generically: No

Controlled Substance: No

Administration Route: Oral

Active Ingredient: Risdiplam

Dosage Form(s): Oral solution

What Is Evrysdi Used For?

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

SMA is a rare genetic disorder estimated to occur in 1 out of 11,000 births. SMA causes the neurons (nerve cells) that control muscles to die and stop sending signals to those muscles. This nerve cell death leads to muscle weakness and wasting.

The severity and age of onset of SMA can vary depending on which form of the disease a person has. Symptoms most commonly occur during infancy and include poor muscle tone, delays in meeting milestones such as sitting, and feeding problems. Other symptoms include:

  • Muscle weakness
  • Muscle wasting
  • Inability to walk
  • Abnormal tongue movements and difficulty swallowing
  • Problems breathing, which may require ventilation (breathing support using a ventilator)

How to Take Evrysdi

Evrysdi is available as an oral solution that's taken by mouth. Evrysdi is typically prescribed once daily and should be taken after a meal, around the same time each day. For breastfed infants, administer Evrysdi after breastfeeding. Do not mix Evrysdi with milk or formula.

You should measure the prescribed dose of Evrysdi with the reusable oral syringe provided with your prescription. Administer Evrysdi immediately after drawing up the dose. If the dose remains in the oral syringe for more than 5 minutes, discard it and prepare a new dose.

After administering Evrysdi, drink some water to ensure the whole dose has been completely swallowed.

Avoid getting Evrysdi on your skin or in your eyes. Wash your skin with soap and water, or rinse your eyes with water if you come into contact with the Evrysdi solution.

Storage

Store Evrysdi in the refrigerator in its original amber bottle to protect it from light. Keep Evrysdi, and all of your medicines, in a safe location, out of the reach of children and pets.

How Long Does Evrysdi Take to Work?

The time it takes Evrysdi to work may be different for everyone. During clinical trials, more infants were able to sit unassisted for at least five seconds after 12 months of treatment with Evrysdi than would be expected for people with SMA without treatment. Older children experienced improvements in their ability to perform certain daily activities after 12 months of treatment. Be sure to talk with your or your child's healthcare provider about what you can expect after starting Evrysdi.

What are the Side Effects of Evrysdi?

This is not a complete list of side effects and others may occur. A healthcare provider can advise you on side effects. If you experience other effects, contact your pharmacist or a healthcare provider. You may report side effects to the Food and Drug Administration (FDA) at fda.gov/medwatch or 800-FDA-1088.

Common Side Effects

You may experience side effects while taking Evrysdi. Be sure to let your healthcare provider know if you develop any side effects that bother you or don't go away.

Common side effects include:

Severe Side Effects

Contact your healthcare provider immediately if you develop any signs of a severe reaction. Call 911 if your symptoms feel life-threatening.

Serious side effects and their symptoms include:

  • An allergic reaction, including a severe form called anaphylaxis: Signs of an allergic reaction include rash, hives, itching, wheezing, trouble breathing, or swelling of the mouth, face, lips, tongue, or throat.
  • Pneumonia: Contact your healthcare provider if you develop shortness of breath or a cough.
  • Urinary tract infection (UTI): Symptoms include blood in the urine, burning or pain while urinating, feeling like you need to urinate often or right away, fever, lower stomach pain, or pelvic pain.

Long-Term Side Effects

Evrysdi may negatively affect male fertility and impact your ability to father a child in the future. Talk with your healthcare provider about this risk. Sperm preservation before starting treatment may be an option for some people.

Report Side Effects

Evrysdi may cause other side effects. Call your healthcare provider if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your healthcare provider may send a report to the FDA's MedWatch Adverse Event Reporting Program or by phone (800-332-1088).

Dosage: How Much Evrysdi Should I Take?

Drug Content Provided and Reviewed by IBM Micromedex®

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

  • For oral dosage form (solution):
    • For spinal muscular atrophy:
      • Adults and children 2 years of age and older weighing 20 kilograms (kg) or more—5 milligrams (mg) once a day.
      • Children 2 years of age and older weighing less than 20 kg—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.25 milligram per kilogram (mg/kg) of body weight once a day.
      • Infants 2 months to less than 2 years of age—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.2 mg/kg of body weight once a day.
      • Infants younger than 2 months of age—Use and dose must be determined by your doctor.

Modifications

The following modifications (changes) should be kept in mind when using Evrysdi:

Pregnancy: In animal studies, Evrysdi was found to cause fetal harm. For this reason, people who can become pregnant must use an effective form of birth control during treatment with Evrysdi and for at least one month after stopping Evrysdi. If you become pregnant, let your healthcare provider know immediately.

Breastfeeding: We don't know enough about the safety of Evrysdi in human breastmilk and nursing babies. If you plan to breastfeed, talk with your healthcare provider about the benefits and risks of taking Evrysdi while nursing and the different ways to feed your baby.

Adults over 65: Clinical studies haven't included a large enough number of people in this age group to see whether they respond differently from younger adults.

Children: Evrysdi is approved to treat SMA children. The safety and effectiveness of using Evrysdi in younger children are not known.

Administration modifications (changes): If you or your child have a feeding tube (nasogastric or gastrostomy tube), Evrysdi may be administered through the tube. Be sure to flush the tube with water after administering the dose to ensure the entire dose gets delivered.

Missed Dose

If you forget to take Evrysdi and it is within six hours, take your dose and resume your regular dosing schedule. If it has been more than six hours since your missed dose, skip the dose and take Evrysdi the next day at your usual time.

If you vomit after taking your dose or don't fully swallow your dose, don't take another dose. Wait until the next day to take Evrysdi at your usual time.

Try to find ways that work for you to help yourself remember to routinely take your medication. If you miss too many doses, Evrysdi might be less effective.

Overdose: What Happens If I Take Too Much Evrysdi?

There is limited information available about an Evrysdi overdose.

If you think that you're experiencing an overdose or life-threatening symptoms, however, seek immediate medical attention.

What Happens If I Overdose on Evrysdi?

If you think you or someone else may have overdosed on Evrysdi, call a healthcare provider or the Poison Control Center (800-222-1222).

If someone collapses or isn't breathing after taking Evrysdi, call 911 immediately.

Precautions

Drug Content Provided and Reviewed by IBM Micromedex®

It is very important that your doctor check your child's progress at regular visits to make sure this medicine is working properly. Blood tests may be needed to check for unwanted effects.

Using this medicine while you are pregnant can harm your unborn baby. If you are a woman who can bear children, your doctor may give you a pregnancy test before you start using this medicine to make sure you are not pregnant. Use an effective form of birth control during treatment and for at least 1 month after the last dose.

If you plan to have children, talk with your doctor before using this medicine. Some men using this medicine have become infertile (unable to have children).

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or non-prescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

What Are Reasons I Shouldn’t Take Evrysdi?

Do not take Evrysdi if you are allergic to risdiplam or any other ingredient in Evrysdi. Ask your pharmacist or healthcare provider for a full list of the ingredients if you're unsure.

What Other Medications Interact With Evrysdi?

Use caution when taking Evrysdi with the following medications:

  • Metformin
  • Tambocor (flecainide)
  • Procainamide
  • Tikosyn (dofetilide)
  • Epivir (lamivudine)
  • Mirapex (pramipexole)

Other medications may interact with Evrysdi. Be sure to inform your healthcare provider and pharmacist about all the medicines you take, including over-the-counter, nonprescription products, vitamins, or herbal medicines.

What Medications Are Similar?

Evrysdi helps treat SMA by increasing the amount of survival motor neuron proteins, a protein that people with SMA struggle to make. Evrysdi is the only oral medication used to treat SMA that works this way. Other non-oral medicines that also increase the amount of survival motor neuron protein include:

  • Sprinraza (nusinersen)
  • Zolgensma (onasemnogene abeparvovec)

Unlike Evrysdi, these medications are administered by injection, either intrathecally (into the space around your spinal cord) for Sprinraza, or intravenously (into your vein) for Zolgensma. These treatments also differ based on how often they are administered, the age group they are approved to treat, and side effects. You and your healthcare provider will work together to choose a treatment plan that works for you.

Frequently Asked Questions

  • What is Evrysdi use for?

    Evrysdi is used to treat spinal muscular atrophy (SMA) in adults and children over 2 months. Evrysdi is not a cure, but it can help improve motor function.

  • How does Evrysdi work?

    People with SMA have low levels of an important protein called survival motor neuron protein. This protein is needed for normal muscle function, and without it, your muscle can weaken and break down. Evrysdi works by increasing your body's production of survival motor neuron protein.

  • What are the side effects of Evrysdi?

    The most common side effects include fever, diarrhea, rash, constipation, vomiting, and signs of the common cold.

How Can I Stay Healthy While Taking Evrysdi?

Living with or caring for a loved one with SMA can have its challenges, but there are ways to improve your quality of life. Refer below for some general tips to support your health:

  • Take SMA-related medications as recommended by your healthcare provider.
  • Consider working with a registered dietitian nutritionist to ensure you are meeting your dietary requirements.
  • Follow-up with all physical therapy appointments, as your healthcare provider recommends, may help influence how SMA progresses.
  • Consider joining a support group to connect with others in the SMA community.

Medical Disclaimer

Verywell Health's drug information is meant for educational purposes only and is not intended to replace medical advice, diagnosis, or treatment from a healthcare provider. Consult your healthcare provider before taking any new medication(s). IBM Watson Micromedex provides some of the drug content, as indicated on the page.

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Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
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