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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. You may also need to take an antibiotic for a period of time.
Even if you receive vaccines for certain 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; 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.
You will be given the Medication Guide. Read the information carefully and ask your doctor or pharmacist if you have any questions. You can also visit http://www.fda.gov/Drugs/DrugSafety/ucm085729.htm to obtain the Medication Guide.
Talk to your doctor about the risks of taking iptacopan.
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) and to decrease protein in the urine in people with a certain type of kidney disease called complement 3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN). 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.
HOW should this medicine be used?
Iptacopan comes as a capsule to take by mouth. Take with or without food twice a day. Take iptacopan at around the same times every day.
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 your condition but does not cure it. Continue to take iptacopan even if you feel well. Do not stop taking iptacopan without talking to your doctor.
Are there OTHER USES for this medication?
This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.
What SPECIAL PRECAUTIONS should I follow?
Before taking iptacopan,
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 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:
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 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 http://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 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 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. http://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 http://goo.gl/c4Rm4p for more information.
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.
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.
This report on medications is for your information only, and is not considered individual patient advice. Because of the changing nature of drug information, please consult your physician or pharmacist about specific clinical use.
The American Society of Health-System Pharmacists, Inc. represents that the information provided hereunder was formulated with a reasonable standard of care, and in conformity with professional standards in the field. The American Society of Health-System Pharmacists, Inc. makes no representations or warranties, express or implied, including, but not limited to, any implied warranty of merchantability and/or fitness for a particular purpose, with respect to such information and specifically disclaims all such warranties. Users are advised that decisions regarding drug therapy are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and the information is provided for informational purposes only. The entire monograph for a drug should be reviewed for a thorough understanding of the drug's actions, uses and side effects. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information is not a substitute for medical care.
AHFS® Patient Medication Information™ Copyright, 2025. The American Society of Health-System Pharmacists®, 4500 East-West Highway, Suite 900, Bethesda, Maryland. All Rights Reserved. Duplication for commercial use must be authorized by ASHP.
Selected Revisions: October 15, 2025.