Brand Name(s): Fabhalta®

Why is this medicine prescribed?

Iptacopan is used to treat paroxysmal nocturnal hemoglobinuria (PNH: a type of anemia in which too many red blood cells are broken down in the body, so there are not enough healthy cells to bring oxygen to all parts of the body). Iptacopan is in a class of medications called complement inhibitors. It works by blocking the activity of the part of the immune system that may damage blood cells.

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?

Iptacopan comes as a capsule to take by mouth. It is usually taken with or without food twice a day. Take iptacopan at around the same times 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 iptacopan exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Swallow the capsules whole; do not split, chew, or break them.

Iptacopan controls PNH but does not cure it. Continue to take iptacopan even if you feel well. Do not stop taking iptacopan without talking to your doctor.

What special precautions should I follow?

Before taking iptacopan,

  • tell your doctor and pharmacist if you are allergic to iptacopan, any other medications, or any of the ingredients in iptacopan capsules. Ask your pharmacist or check the Medication Guide 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. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
  • tell your doctor if you have or have ever had high blood cholesterol, high blood fats (triglycerides), or kidney or liver problems.
  • tell your doctor if you are pregnant or plan to become pregnant, or are breast-feeding. If you become pregnant while taking iptacopan, call your doctor.
  • tell your doctor if you are breastfeeding or plan to breastfeed. You should not breastfeed while taking iptacopan and for 5 days after your final dose.
  • you should know that your condition may cause too many red blood cells to break down after you stop taking iptacopan. This could result in blood clots, stroke, or heart attack. Your doctor will monitor you carefully and may order laboratory tests for at least 2 weeks after you finish your treatment. Call your doctor right away if you develop any of the following symptoms: extreme tiredness, blood in urine, stomach pain, difficulty swallowing, shortness of breath, or an inability to get or keep an erection.

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, even it if it almost time for the next dose, and then continue your regular dosing schedule.

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 side effects can this medicine cause?

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

  • headache
  • runny nose, nasal congestion, sneezing, sore throat, or cough
  • diarrhea
  • nausea
  • joint pain
  • rash

Some side effects can be serious. If you experience any of the symptoms listed in the IMPORTANT WARNING or SPECIAL PRECAUTIONS sections, call your doctor immediately or get emergency medical treatment.

Iptacopan 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).

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 other information should I know?

Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to iptacopan.

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.

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