Iptacopan

Iptacopan may increase the risk that you will develop a meningococcal infection (an infection that may affect the covering of the brain and spinal cord and may spread through the bloodstream) or other serious infections during your treatment or for some time afterward. These infections may cause death in a short period of time. You will need to receive certain vaccines at least 2 weeks before you begin your treatment with iptacopan to decrease the risk that you will develop this type of infection. If you have received these vaccines in the past, you may need to receive a booster dose before you begin your treatment. If your doctor feels that you need to begin treatment with iptacopan right away, you will receive your vaccines as soon as possible but will need to take an antibiotic for a period of time.

Even if you receive vaccines for certain bacterial infections, there is still a risk that you may develop serious infections during or after your treatment with iptacopan. If you experience any of the following symptoms, call your doctor immediately or get emergency medical help: headache that comes along with nausea or vomiting, fever, a stiff neck, or a stiff back; fever of 103°F (39.4°C) or higher; rash and fever; fever with chest pain, rapid heartbeat, cough, shortness of breath, or fast breathing; confusion; muscle aches and other flu-like symptoms; or if your eyes are sensitive to light.

Tell your doctor if you have fever or other signs of infection before you begin your treatment with iptacopan. Your doctor may tell you not to take iptacopan if you have a certain type of infection.

Your doctor will give you a patient safety card with information about the risk of developing serious infections during or for a period of time after your treatment. Carry this card with you at all times during your treatment and for 2 weeks after your treatment. Show the card to all healthcare providers who treat you so that they will know about your risk.

A program called Fabhalta REMS has been set up to decrease the risks of taking iptacopan. You will only be able to take iptacopan if the doctor who prescribes your medication is enrolled in the program, has talked to you about the risks of serious infections, has given you a patient safety card, and has made sure that you received certain vaccinations. You can also only receive the medication from a pharmacy that participates in the program. Ask your doctor if you have any questions about participating in the program or how to get your medication.

Your doctor or pharmacist will give you the manufacturer's patient information sheet (Medication Guide) when you begin treatment with iptacopan and each time you refill your prescription. Read the information carefully and ask your doctor or pharmacist if you have any questions. You can also visit the Food and Drug Administration (FDA) website (http://www.fda.gov/Drugs/DrugSafety/ucm085729.htm) or the manufacturer's website to obtain the Medication Guide.

Talk to your doctor about the risks of taking iptacopan.

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.

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.

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

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.

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

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.

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).

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.

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.

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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