Risdiplam

Risdiplam is used to treat a genetic condition called spinal muscular atrophy in children and adults.

Risdiplam may also be used for reasons not mentioned in this medication guide.

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

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

You may take risdiplam with or without food, but take it at the same time each day.

Swallow the tablet whole with water and do not crush, chew, or break it.

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

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

Read and carefully follow the Instructions for Use provided with your medicine. Ask your doctor or pharmacist if you do not understand these instructions.

If you are breastfeeding a child using liquid medicine, give this medicine to a child before or after feeding. Do not mix risdiplam liquid with infant formula or milk.

If the entire dose is not swallowed or if you vomit shortly after taking risdiplam, do not take another dose. Take your next dose as scheduled.

If you are unable to swallow the tablet whole, read and carefully follow the instructions provided to you on how to prepare and take risdiplam. Risdiplam liquid may also be given through a feeding tube. Ask a doctor or pharmacist if you do not understand these instructions.

Do not change your dose or stop using a medicine without your doctor's advice.

Do not use liquid medicine in powder form. If you receive the medicine as a powder, ask your pharmacist to mix the medicine for you.

Store the tablet in the original container tightly closed at room temperature, away from moisture and heat. Ask your pharmacist how to properly store the liquid medicine and the reusable oral syringes.

Use only as directed. Inform your doctor if you are taking other medicines or have other medical conditions or allergies.

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

May harm an unborn baby. You may need to take a negative pregnancy test before starting this treatment. Use effective birth control while using risdiplam and for at least 1 month after your last dose. Inform your doctor if you are pregnant or plan to become pregnant.

It may be more difficult 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.

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

Serious side effects may happen more often, or you may have none at all.

Common side effects of risdiplam may include:

• fever, cough, or lung infection;
• 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.

Usual Adult Dose for Spinal Muscular Atrophy:

5 mg orally once a dayComments:-See Other Comments or 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:

Less than 2 months: 0.15 mg/kg orally once a day2 months to less than 2 years: 0.2 mg/kg orally once a day2 years and older:-Weight less than 20 kg: 0.25 mg/kg orally once a day-Weight 20 kg or more: 5 mg orally once a dayComments:-See Other Comments or Administration Advice for important information on dose preparation and administration.Use: For the treatment of spinal muscular atrophy

Take the medicine as soon as possible, but skip the missed dose if you are more than 6 hours late. Do not take two doses at once.

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

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

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