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Generic name: risdiplam [ ris-DIP-lam ]
Brand name: Evrysdi
Dosage form: oral powder for reconstitution (0.75 mg/mL)
Drug class: Miscellaneous uncategorized agents

Medically reviewed by on Jan 23, 2024. Written by Cerner Multum.

What is risdiplam?

Risdiplam is used to treat a genetic condition called spinal muscular atrophy (weakness and wasting of the muscles needed for movement of the body) in children and adults.

Risdiplam may also be used for purposes not listed in this medication guide.

Risdiplam side effects

Get emergency medical help if you have signs of an allergic reaction: hives, difficult breathing, swelling of your face, lips, tongue, or throat.

Risdiplam may cause serious side effects. Call your doctor at once if you have:

Common side effects of risdiplam may include:

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.


Use only as directed. Tell your doctor if you use other medicines or have other medical conditions or allergies.

Before taking this medicine

Tell your doctor if you have or have ever had liver problems.

You may need to have a negative pregnancy test before starting this treatment.

If you are pregnant, your name may be listed on a pregnancy registry to track the effects of risdiplam on the baby.

May harm an unborn baby. Use effective birth control while using risdiplam and for at least 1 month after your last dose. Tell your doctor if you become pregnant.

It may be harder for you to get a woman pregnant while you are using risdiplam.

Ask a doctor if it is safe to breastfeed while using this medicine.

How should I take risdiplam?

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

Risdiplam is supplied as a liquid that must be mixed at the pharmacy. Do not use this medicine in powder form. If you receive the medicine as a powder, ask your pharmacist to mix the medicine for you.

Doses are based on age and weight. Your dose may change if you gain or lose weight.

Always take this medicine after a meal, at the same time each day.

To make sure you swallow the entire dose, drink a glass of water after taking the medicine.

If you are breastfeeding a child using risdiplam, give the medication to the child after feeding. Do not mix risdiplam with infant formula or milk.

Measure liquid medicine with the supplied measuring device (not a kitchen spoon).

Prepare a dose only when you are ready to give it. After placing risdiplam into the oral syringe, you must give the medicine within 5 minutes. If not given within 5 minutes, throw out the liquid and prepare a new dose.

If the entire dose is not swallowed or if you vomit shortly after taking risdiplam, do not take another dose. Wait until your next scheduled dose time to take the medicine again.

If you are giving this medicine through a nasogastric or gastrostomy tube, your doctor should first check the tube. Follow all directions about how to give risdiplam through the tube, and flush the tube with water after each dose.

The oral syringe is reusable. Wash the syringe after each use and allow it to air dry. If your syringe is lost or damaged, call your pharmacist for a new one.

Do not stop taking risdiplam or change your dose without your doctor's advice.

Store mixed medicine in the refrigerator in the original container and protect from light. Do not freeze.

You may store the medicine for a total combined period of 5 days at room temperature before returning it to the refrigerator or up to 64 days in a refrigerator.

Risdiplam dosing information

Usual Adult Dose for Spinal Muscular Atrophy:

5 mg orally once a day

-See Other Comments/Administration Advice for important information on dose preparation and administration.

Use: For the treatment of spinal muscular atrophy.

Usual Pediatric Dose for Spinal Muscular Atrophy:

2 months to less than 2 years: 0.2 mg/kg orally once a day

2 years and older weight less than 20 kg: 0.25 mg/kg orally once a day

2 years and older weight 20 kg or more: 5 mg orally once a day

-See Other Comments/Administration Advice for important information on dose preparation and administration.

Use: For the treatment of spinal muscular atrophy in patients 2 months of age and older.

What happens if I miss a dose?

Use the medicine as soon as you can, but skip the missed dose if you are more than 6 hours late for the dose. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking risdiplam?

Avoid getting risdiplam in your eyes or on your skin. If this happens, wash your skin with soap and water or rinse your eyes with water.

What other drugs will affect risdiplam?

Other drugs may affect risdiplam, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.