Iptacopan is used in adults to treat a disease called paroxysmal nocturnal hemoglobinuria (PNH).

Iptacopan is available only under a special program and from a certified pharmacy. You must understand the risks and benefits of this medicine.

Iptacopan may also be used for purposes not listed in this medication guide.

Before you start using iptacopan, tell your doctor if you have any signs of infection such as fever, chills, cough, body aches, tiredness, open sores or skin wounds, diarrhea, stomach pain, weight loss, painful urination, or coughing up blood.

You should not use iptacopan if you are allergic to it, or if you have:

• a serious infection caused by certain types of bacteria such as meningitis, pneumonia, or a bloodstream infection.

Tell your doctor if you have or have ever had:

• a chronic or recent infection;
• fever;
• high cholesterol or triglycerides;
• recently received a vaccine or are scheduled to receive a vaccine; or
• liver or kidney disease.

Make sure you are current on all vaccines that protect against serious bacterial infections at least 2 weeks before you start using iptacopan. If you were vaccinated in the past, you may need additional vaccinations or booster doses.

It is not known if iptacopan will harm an unborn baby. However, having paroxysmal nocturnal hemoglobinuria (PNH) during pregnancy may cause complications or medical problems in both mother and baby. The benefit of treating this condition may outweigh any risks to the baby.

Do not breastfeed while using this medicine, and for at least 5 days after your last dose.

Use Fabhalta (Oral) exactly as directed on the label, or as prescribed by your doctor. Do not use in larger or smaller amounts or for longer than recommended.

Get emergency medical help if you have signs of an allergic reaction: hives, difficult breathing, swelling of your face, lips, tongue, or throat.

You may get infections more easily, even serious or fatal infections. Call your doctor right away if you have signs of infection such as:

• fever, chills, sore throat;
• mouth sores, red or swollen gums;
• pale skin, easy bruising, unusual bleeding;
• chest discomfort, wheezing, dry cough or hack, rapid weight loss; or
• symptoms of meningitis--headache, neck stiffness, increased sensitivity to light, nausea, vomiting, confusion, drowsiness.

Your condition may get worse if you stop taking this medicine. If you stop using iptacopan, tell your doctor if you have any new or worsening symptoms, such as tiredness, confusion, stomach pain, trouble breathing or swallowing, blood in your urine, difficulty having an erection, chest pain, pain in one leg, or numbness or weakness on one side of the body.

Common side effects may include:

• headache;
• infections caused by bacteria or virus;
• cold symptoms such as stuffy nose, sneezing, sore throat;
• rash; or
• diarrhea, nausea, stomach pain.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.