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

Risdiplam is for use in adults and children at least 2 months old.

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

Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

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

A healthcare provider will teach you how to properly prepare the daily dose. Read and carefully follow any Instructions for Use provided with your medicine.

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.

Measure risdiplam carefully. Use only one of the dosing syringes provided (choose the syringe size for your correct dose). Do not use a kitchen spoon or any other medicine dose-measuring device.

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.

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

If the child using risdiplam is breastfed, give the medicine after feeding. Do not mix risdiplam with infant formula or milk.

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

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.

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

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.

Store risdiplam in the original bottle in an upright position in the refrigerator. Protect from light and do not freeze. Keep the bottle tightly closed when not in use. Throw away any unused liquid if it has been longer than 64 days since it was mixed at the pharmacy.

Risdiplam doses are based on weight in children. Your child's dose needs will change as the child grows and gains weight.

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

Usual Adult Dose for Spinal Muscular Atrophy:

5 mg orally once a day

Comments:
-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

Comments:
-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.

Risdiplam is not approved for use in a child younger than 2 months old.

Tell your doctor if you have ever had liver problems.

You may need to have a negative pregnancy test before starting this treatment. Tell your doctor if you plan to become pregnant.

Risdiplam may harm an unborn baby. Use effective birth control to prevent pregnancy while you are using risdiplam and for at least 1 month after your last dose. Tell your doctor right away if you become pregnant.

Risdiplam may affect fertility (ability to have children), in men. Talk to your doctor if you have concerns about this risk.

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

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

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

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:

• pain or burning when you urinate; or

• signs of a lung infection--fever, cough with mucus, chest pain, feeling short of breath.

Common side effects of risdiplam may include:

• fever, lung infection;

• nausea, vomiting;

• diarrhea, constipation;

• rash; or

• cold symptoms such as stuffy nose, sneezing, sore throat.

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.

Other drugs may affect risdiplam, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.