Risdiplam

WHY is this medicine prescribed?

Risdiplam is used for the treatment of spinal muscular atropy (SMA; an inherited condition that affected the nerves and causes muscle weakness). Risdiplam is in a class of medications called survival of motor neuron 2 (SMN2) splicing modifier. It works by increasing the amount of a protein in the body's nervous system to improve muscle strength and movement.

Are there OTHER USES for this medicine?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

HOW should this medicine be used?

Risdiplam comes as a tablet and solution (liquid) to take by mouth. Take once daily with or without food. Take risdiplam at around the same time every day.

Take risdiplam exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Swallow the tablets whole; do not split, chew, or crush them.

If you are unable to swallow the tablet, it may be placed in one teaspoonful (5 mL) of non-chlorinated drinking water in a small cup. Do not use any other liquids to prepare. Swirl the mixture for 3 minutes until fully mixed; some particles will remain. Refill the cup with 1 tabletspoon (15 mL) of non-chlorinated drinking water, swirl, and drink right away. Use the mixture right after preparation and do not expose to sunlight. If the mixture is not given within 10 minutes, discard it and prepare a new dose.

The solution will be prepared by your pharmacist. Use an oral syringe (measuring device) to measure the dose. Give the solution within 5 minutes after it is drawn into the syringe. If it is not given within 5 minutes, discard and prepare a new dose. After the dose is swallowed, drink water to be sure the entire dose is taken.

If your child is a breastfed infant, you can give the dose of solution before or after breastfeeding, but do not mix with formula or milk.

Risdiplam solution can be given into a nasogastric or gastrostomy tube. Flush with water after giving the dose.

Do not take another dose of risdiplam if the tablet or solution is not fully swallowed or if vomiting occurs after taking a dose. Resume your regular dosing at the next scheduled time.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

What SPECIAL PRECAUTIONS should I follow?

Before taking risdiplam,

• tell your doctor or pharmacist if you are allergic to this medication, any part of this medication, or any other medications, foods or substances. Tell your doctor or pharmacist about the allergy and what symptoms you had.

• some medications should not be taken with risdiplam. Other medications may cause dosing changes or extra monitoring when taken with risdiplam. Make sure you have discussed any medications you are currently taking or plan to take before starting risdiplam with your doctor and pharmacist. Before starting, stopping, or changing any medications while taking risdiplam, please get the advice of your doctor or pharmacist.

• tell your doctor if you have or have ever had any medical conditions.

• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. If you become pregnant while taking risdiplam, call your doctor immediately. Risdiplam may harm the fetus.

  You may be required to have a pregnancy test prior to starting risdiplam. You should use birth control during and for at least one month after treatment with risdiplam. Talk to your doctor about what birth control is right for you.

  You may develop a lower sperm count and fertility problems after you take risdiplam. If you want to have children, discuss your options with a healthcare provider before treatment.

What SPECIAL DIETARY instructions should I follow?

Unless your doctor tells you otherwise, continue your normal diet.

What should I do IF I FORGET to take a dose?

If it is within 6 hours of the scheduled time, take the missed dose as soon as you remember. However, if it is 6 hours or greater from the scheduled time, skip the missed dose and continue your regular dosing schedule. Do not take a double dose to make up for a missed one.

What SIDE EFFECTS can this medicine cause?

Risdiplam may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• fever
• rash
• diarrhea, constipation, vomiting
• runny nose, sneezing, cough, sore throat

Risdiplam may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What should I know about STORAGE and DISPOSAL of this medication?

Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it away from excess heat and moisture (not in the bathroom).

Keep the solution in the refrigerator in the bottle provided. Protect from light and keep tightly closed. Do not freeze. Dispose of any unused solution after 64 days. If a refrigerator is not available, risdiplam solution can be kept at room temperature for 5 days. The solution may be returned to the refrigerator, but the total time at room temperature cannot exceed 5 days.

Store the tablets at room temperature.

Keep all medication out of sight and reach of children as many containers are not child-resistant. Always lock safety caps. Place the medication in a safe location – one that is up and away and out of their sight and reach. https://www.upandaway.org

Dispose of unneeded medications in a way so that pets, children, and other people cannot take them. Do not flush this medication down the toilet. Use a medicine take-back program. Talk to your pharmacist about take-back programs in your community. Visit the FDA's Safe Disposal of Medicines website https://goo.gl/c4Rm4p for more information.

What should I do in case of OVERDOSE?

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

What OTHER INFORMATION should I know?

Keep all appointments with your doctor.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

Keep a written list of all of the prescription and nonprescription (over-the-counter) medicines, vitamins, minerals, and dietary supplements you are taking. Bring this list with you each time you visit a doctor or if you are admitted to the hospital. You should carry the list with you in case of emergencies.