risdiplam

What is the most important information I should know about risdiplam?

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.

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).

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.

What should I discuss with my healthcare provider before taking risdiplam?

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

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.

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.

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.

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.

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.

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.

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. Go back to the regular schedule the following day. Do not use two doses at one time.

What happens if I overdose?

What should I avoid while taking risdiplam?

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

What are the possible side effects of risdiplam?

• 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 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.

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 your current medicines and any medicine you start or stop using.

Where can I get more information?

Your pharmacist can provide more information about risdiplam.