Maralixibat

WHY is this medicine prescribed?

Maralixibat is used to treat itching caused by Alagille syndrome (ALGS, an inherited condition in which bile builds up in the liver and causes liver damage) in adults and children 1 year of age or older. Maralixibat is in a class of medications called ileal bile acid transporter inhibitors. It works by decreasing the amount of bile acids in the body.

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?

Maralixibat comes as a solution (liquid) to take by mouth. It is usually taken once a day, thirty minutes before the first meal of the day. Take maralixibat at around the same time every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take maralixibat exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Your doctor may start you on a low dose of maralixibat and increase your dose after one week.

Use an oral syringe (measuring device) to accurately measure and take your dose of maralixibat solution. Ask your pharmacist for an oral syringe if one is not included with your medication. Do not use a household teaspoon to measure the solution. Replace each oral syringe after using it for 30 days or if it becomes damaged or hasn't been cleaned. Follow the manufacturer's instructions about how to clean the oral syringe. Ask your doctor or pharmacist if you if you have any questions.

Follow these steps to measure the dose using the syringe provided:

1. Open the bottle by pushing down on the cap and turning the cap at the same time.
2. Push the plunger of the measuring device completely down to the tip.
3. Insert the tip of the measuring device firmly into the opening on the top of the bottle.
4. Turn the bottle (with the measuring device attached) upside down.
5. Pull back on the plunger slowly until the amount of liquid prescribed by your doctor fills the measuring device to the appropriate marking. If you are not sure how to correctly measure the dose your doctor has prescribed, ask your doctor or pharmacist.
6. Turn the bottle (with the measuring device attached) right-side up and slowly remove the measuring device.
7. Take maralixibat directly into your mouth from the measuring device; you should do this while sitting or standing.
8. Replace the cap on the bottle and close tightly.
9. Follow the manufacturer's instructions about how to clean the oral syringe.
10. Call your doctor or pharmacist to find out how you should measure a dose of maralixibat solution if you do not have the measuring device that came with this medication.

Maralixibat controls itching caused by Alagille syndrome but does not cure it. Continue to take maralixibat even if you feel well. Do not stop taking maralixibat without talking to your doctor.

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

What SPECIAL PRECAUTIONS should I follow?

Before taking maralixibat,

• tell your doctor and pharmacist if you are allergic to maralixibat, any other medications, or any of the ingredients in maralixibat oral solution. Ask your pharmacist for a list of the ingredients.

• tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take while taking maralixibat. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.

• if you are taking cholestyramine (Prevalite), colesevelam (Welchol), or colestipol (Colestid), take them at least 4 hours before or 4 hours after maralixibat.

• tell your doctor if you have or have ever had any blood clotting problems; malabsorption problems (problems absorbing food or vitamins); osteoporosis (a condition in which the bones are fragile and break easily), osteomalacia (weak bones due to a lack of vitamin D or difficulty absorbing this vitamin), or other conditions that cause weak bones; or any liver problems, including hepatitis or cirrhosis.

• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. If you become pregnant while taking maralixibat, call your doctor.

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?

Take the missed dose as soon as you remember it. However, if it is more than 12 hours since you were to take the dose, skip the missed dose and continue with your regular dosing schedule. Do not take a double dose to make up for a missed one.

What SIDE EFFECTS can this medicine cause?

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

• diarrhea
• stomach pain
• nausea

Maralixibat may cause side effects. Some side effects can be serious. If you experience any of these symptoms call your doctor immediately:

• vomiting
• fever
• skin or the whites of eyes turn yellow
• dark or brown urine
• pain on the right side of the abdomen
• loss of appetite
• vomiting blood
• bloody or black, tarry stools

Taking maralixibat may increase the risk that you will experience a bone fracture. Talk to your doctor about the risk of taking this medication and about ways to keep your bones healthy during your treatment.

Maralixibat 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 (http://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 at room temperature and away from excess heat and moisture (not in the bathroom).

Keep this medication closed tightly and dispose of any unused medication 45 days after first opening the container.

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. http://www.upandaway.org

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (http://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

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 or laboratory. Your doctor will order certain lab tests before and during your treatment to check your body's response to maralixibat.

Before having any laboratory test, tell your doctor and the laboratory personnel that you are taking maralixiabat.

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

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.