Pegcetacoplan is used to treat paroxysmal nocturnal hemoglobinuria (PNH) in adults.

Pegcetacoplan is available only under a special program. You must be registered in the program and understand the risks and benefits of pegcetacoplan.

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

You may get infections more easily, even serious or fatal infections. Call your doctor if you have a fever, flu-like symptoms, muscle pain, headache, confusion, neck or back stiffness, vomiting, rash, or your eyes are more sensitive to light.

You will need to receive certain vaccinations before you start using pegcetacoplan.

Read the Patient Safety Card about serious infections and the symptoms to watch for. Keep the card with you at all times while using pegcetacoplan and for at least 2 months after your last dose. Your infection risk could last for several weeks after you stop using pegcetacoplan.

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

If you've been using another drug called ravulizumab (Ultomiris), you will need to wait 4 weeks after your last dose of ravulizumab before starting treatment with pegcetacoplan.

If you've been using another drug called eculizumab (Soliris), keep using it for 4 weeks after your first dose of pegcetacoplan and then stop the eculizumab.

Pegcetacoplan is injected under your skin with an infusion pump, usually twice per week or once every 3 days. You may be able to give this injection to yourself.

Read and follow all Instructions for Use. Ask your doctor or pharmacist if you need help.

Prepare an injection only when you are ready to give it. Call your pharmacist if the medicine looks cloudy, has changed colors, or has particles in it.

Do not reuse a needle or syringe. Place them in a puncture-proof "sharps" container and dispose of it following state or local laws. Keep out of the reach of children and pets.

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

Pegcetacoplan can have long lasting effects on your body, even after you stop using this medicine. If you have PNH and you stop using pegcetacoplan, your doctor may need to check your progress for at least 8 weeks after your last dose.

Read the Patient Safety Card about serious infections and the symptoms to watch for. Keep the card with you at all times while using pegcetacoplan and for at least 2 months after your last dose. Your infection risk could last for several weeks after you stop using pegcetacoplan.

This medicine can affect the results of certain medical tests. Tell any doctor who treats you that you are using pegcetacoplan.

Store vials in the refrigerator, in the original carton to protect from light. Do not use expired pegcetacoplan.

Usual Adult Dose for Paroxysmal Nocturnal Hemoglobinuria:

Subcutaneous: 1,080 mg twice weekly infused via a commercially available infusion pump with a reservoir of at least 20 mL

To reduce the risk of hemolysis with abrupt treatment discontinuation when switching from C5 inhibitors:
-For patients switching from eculizumab, initiate this drug while continuing eculizumab at its current dose. After 4 weeks, discontinue eculizumab before continuing on monotherapy with this drug
-For patients switching from ravulizumab, initiate this drug no more than 4 weeks after the last dose of ravulizumab

Comments:
-Vaccinate against encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B at least 2 weeks prior to initiating therapy according to current ACIP guidelines.
-It is recommended to provide 2 weeks of antibacterial drug prophylaxis if this drug must be initiated immediately and vaccines are administered less than 2 weeks before starting therapy.

Use: For the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH)

You should not be treated with pegcetacoplan if you are allergic to it, if you have a serious infection (such as pneumonia, meningitis or influenza type B), or if you are not currently vaccinated against these diseases (unless the risks of delaying treatment outweigh the risks of developing an infection).

At least 2 weeks before you start using pegcetacoplan, you will need to receive vaccines to protect against meningococcal infections, pneumonia, and influenza type B (Hib). Even if you've received these vaccinations in the past, you may need them again.

If you need to start receiving pegcetacoplan before you are vaccinated, you may be given antibiotic medicine to take during the first 2 weeks of pegcetacoplan treatment.

Tell your doctor if you have recently had any symptoms of infection (fever, chills, or flu-like symptoms).

May harm an unborn baby. You may need a pregnancy test to make sure you are not pregnant. Use birth control while using pegcetacoplan and for at least 40 days after your last dose. Tell your doctor if you become pregnant.

Do not breastfeed while using this medicine, and for at least 40 days after your last dose.

Not approved for use by anyone younger than 18 years old.

Use the medicine as soon as you can, but skip the missed dose if it is almost time for your next dose. Do not use two doses at one time.

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

Avoid injecting into skin that is red, bruised, hard, tender, injured, or irritated. Do not inject pegcetacoplan into skin areas with scars, stretch marks, or tattoos.

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

Some side effects may occur during the infusion. Tell your caregiver if you feel light-headed or if you have chest pain, trouble breathing, or swelling in your face.

Call your doctor right away if you have signs of infection such as:

• muscle pain with flu-like symptoms (fever, chills);

• fever and a rash or headache;

• headache with nausea and vomiting or stiffness in your neck or back;

• clammy skin;

• extreme pain;

• fast heartbeats, shortness of breath;

• confusion; or

• your eyes may be more sensitive to light.

If you stop using pegcetacoplan, 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, (in men) trouble having an erection, chest pain, pain in one leg, or numbness or weakness on one side of the body.

Common side effects of pegcetacoplan may include:

• stomach pain, vomiting, diarrhea;

• cold sores;

• cold symptoms such as stuffy nose, sneezing, sore throat, cough;

• tiredness; or

• pain, itching, warmth, redness, bruising, bleeding, swelling, or a hard lump where the medicine was injected.

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.

Other drugs may affect pegcetacoplan, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.