Danicopan is used together with eculizumab or ravulizumab to treat the breakdown of red blood cells that occurs outside of blood vessels (extravascular hemolysis), in adults with a disease called paroxysmal nocturnal hemoglobinuria (PNH).

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

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

Before you start using danicopan, 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 danicopan 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;
• recently received a vaccine or are scheduled to receive a vaccine; or
• liver disease.

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

It is not known if danicopan 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 3 days after your last dose.

Use Danicopan (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. Tell your doctor if you have symptoms of low red blood cells (anemia) such as pale skin, tiredness, feeling light-headed or short of breath, cold hands and feet.

Your treatment may be delayed or permanently discontinued if you have certain side effects.

Common side effects may include:

• abnormal liver function tests;
• high cholesterol; or
• headache.

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.