Danicopan

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

Danicopan is available only through a special program and from a certified pharmacy. You must understand the risks and benefits of danicopan.

Danicopan may also be used for reasons not mentioned in this medication guide.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

Read the Patient Wallet Card about serious side effects and learn what symptoms to watch for. Keep the card with you at all times, and for at least 1 week after your last dose.

You will need to take more than one tablet to receive your total dose of danicopan. Follow your doctor's dosing instructions very carefully.

Danicopan is usually taken three times a day, with or without food. Take it the same way each time.

You may be given other medications to help treat or prevent a serious infection or certain side effects. Keep taking these medicines for as long as your doctor has prescribed.

You may get infections more easily, even serious or fatal infections. You will need frequent medical tests.

You will need frequent blood tests to check your liver function before and during treatment with danicopan. You will also need medical tests to check your cholesterol during your treatment.

Your condition may get worse if you change your dose or stop using danicopan suddenly. Ask your doctor before stopping the medicine. Your doctor will watch you closely for at least 2 weeks if you stop taking danicopan.

Store this medicine in the original container at room temperature away from moisture and heat.

You may become more susceptible to infections, including serious or fatal ones. Contact your doctor if you experience flu symptoms, a cough, night sweats, neck stiffness, confusion, or vision issues.

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 danicopan, and for at least 3 days after your last dose.

Get emergency medical help if you have signs of an allergic reaction: hives, difficulty 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 hacking, 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 stopped if you have certain side effects.

Common side effects of danicopan 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.

Usual Adult Dose for Paroxysmal Nocturnal Hemoglobinuria:

Initial dose: 150 mg orally 3 times a day Maintenance dose: May increase to 200 mg orally 3 times a day based on the following parameters:-If hemoglobin (Hgb) level has not increased greater than 2 g/dL after 4 weeks of therapy, OR-If a transfusion was required during the previous 4 weeks, OR -To achieve an appropriate Hgb response based on clinical judgement.Comments:-Limitations of use: This drug has not been shown to be effective as monotherapy and should only be prescribed as an add-on treatment.-Vaccination against encapsulated bacteria should be completed at least 2 weeks prior to starting therapy.Use: As add-on therapy with ravulizumab or eculizumab, for the treatment of extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH)

Take the medicine as soon as you can, but if your next dose is due within 3 hours, skip the missed dose and return to your regular schedule. Do not take two doses at one time.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

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

Other drugs may affect danicopan, including prescription and over-the-counter medicines, vitamins, and herbal products. Inform your doctor about all other medicines you are taking.